KR102271292B1 - Rna-안내 게놈 편집의 특이성을 증가시키기 위한 rna-안내 foki 뉴클레아제(rfn)의 용도 - Google Patents

Rna-안내 게놈 편집의 특이성을 증가시키기 위한 rna-안내 foki 뉴클레아제(rfn)의 용도 Download PDF

Info

Publication number
KR102271292B1
KR102271292B1 KR1020217002429A KR20217002429A KR102271292B1 KR 102271292 B1 KR102271292 B1 KR 102271292B1 KR 1020217002429 A KR1020217002429 A KR 1020217002429A KR 20217002429 A KR20217002429 A KR 20217002429A KR 102271292 B1 KR102271292 B1 KR 102271292B1
Authority
KR
South Korea
Prior art keywords
artificial sequence
dna
lys
leu
description
Prior art date
Legal status (The legal status is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the status listed.)
Active
Application number
KR1020217002429A
Other languages
English (en)
Korean (ko)
Other versions
KR20210013304A (ko
Inventor
제이. 케이쓰 중
솅다르 차이
Original Assignee
더 제너럴 하스피탈 코포레이션
Priority date (The priority date is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the date listed.)
Filing date
Publication date
Family has litigation
First worldwide family litigation filed litigation Critical https://patents.darts-ip.com/?family=51537665&utm_source=google_patent&utm_medium=platform_link&utm_campaign=public_patent_search&patent=KR102271292(B1) "Global patent litigation dataset” by Darts-ip is licensed under a Creative Commons Attribution 4.0 International License.
Application filed by 더 제너럴 하스피탈 코포레이션 filed Critical 더 제너럴 하스피탈 코포레이션
Publication of KR20210013304A publication Critical patent/KR20210013304A/ko
Application granted granted Critical
Publication of KR102271292B1 publication Critical patent/KR102271292B1/ko
Active legal-status Critical Current
Anticipated expiration legal-status Critical

Links

Images

Classifications

    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/0004—Oxidoreductases (1.)
    • C12N9/0071—Oxidoreductases (1.) acting on paired donors with incorporation of molecular oxygen (1.14)
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/87—Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation
    • C12N15/90—Stable introduction of foreign DNA into chromosome
    • C12N15/902—Stable introduction of foreign DNA into chromosome using homologous recombination
    • C12N15/907—Stable introduction of foreign DNA into chromosome using homologous recombination in mammalian cells
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/005—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from viruses
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/195—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from bacteria
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/01—Preparation of mutants without inserting foreign genetic material therein; Screening processes therefor
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/10—Processes for the isolation, preparation or purification of DNA or RNA
    • C12N15/102—Mutagenizing nucleic acids
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/10—Processes for the isolation, preparation or purification of DNA or RNA
    • C12N15/102—Mutagenizing nucleic acids
    • C12N15/1031—Mutagenizing nucleic acids mutagenesis by gene assembly, e.g. assembly by oligonucleotide extension PCR
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/10—Transferases (2.)
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/10—Transferases (2.)
    • C12N9/1003—Transferases (2.) transferring one-carbon groups (2.1)
    • C12N9/1007—Methyltransferases (general) (2.1.1.)
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/14—Hydrolases (3)
    • C12N9/16—Hydrolases (3) acting on ester bonds (3.1)
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/14—Hydrolases (3)
    • C12N9/16—Hydrolases (3) acting on ester bonds (3.1)
    • C12N9/22—Ribonucleases [RNase]; Deoxyribonucleases [DNase]
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/96—Stabilising an enzyme by forming an adduct or a composition; Forming enzyme conjugates
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12Y—ENZYMES
    • C12Y301/00—Hydrolases acting on ester bonds (3.1)
    • C12Y301/21—Endodeoxyribonucleases producing 5'-phosphomonoesters (3.1.21)
    • C12Y301/21004—Type II site-specific deoxyribonuclease (3.1.21.4)
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2319/00—Fusion polypeptide
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2319/00—Fusion polypeptide
    • C07K2319/01—Fusion polypeptide containing a localisation/targetting motif
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2319/00—Fusion polypeptide
    • C07K2319/80—Fusion polypeptide containing a DNA binding domain, e.g. Lacl or Tet-repressor
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/20—Type of nucleic acid involving clustered regularly interspaced short palindromic repeats [CRISPR]
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2710/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA dsDNA viruses
    • C12N2710/00011—Details
    • C12N2710/00033—Use of viral protein as therapeutic agent other than vaccine, e.g. apoptosis inducing or anti-inflammatory
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2770/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssRNA viruses positive-sense
    • C12N2770/00011—Details
    • C12N2770/00033—Use of viral protein as therapeutic agent other than vaccine, e.g. apoptosis inducing or anti-inflammatory
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2800/00—Nucleic acids vectors
    • C12N2800/80—Vectors containing sites for inducing double-stranded breaks, e.g. meganuclease restriction sites
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12Y—ENZYMES
    • C12Y114/00—Oxidoreductases acting on paired donors, with incorporation or reduction of molecular oxygen (1.14)
    • C12Y114/11—Oxidoreductases acting on paired donors, with incorporation or reduction of molecular oxygen (1.14) with 2-oxoglutarate as one donor, and incorporation of one atom each of oxygen into both donors (1.14.11)
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12Y—ENZYMES
    • C12Y201/00—Transferases transferring one-carbon groups (2.1)
    • C12Y201/01—Methyltransferases (2.1.1)
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12Y—ENZYMES
    • C12Y301/00—Hydrolases acting on ester bonds (3.1)

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Organic Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Biomedical Technology (AREA)
  • Biotechnology (AREA)
  • Molecular Biology (AREA)
  • Biochemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Microbiology (AREA)
  • Biophysics (AREA)
  • Medicinal Chemistry (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Crystallography & Structural Chemistry (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Virology (AREA)
  • Cell Biology (AREA)
  • Mycology (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Peptides Or Proteins (AREA)
  • Enzymes And Modification Thereof (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Measuring Or Testing Involving Enzymes Or Micro-Organisms (AREA)
  • Preparation Of Compounds By Using Micro-Organisms (AREA)
  • Developing Agents For Electrophotography (AREA)
  • Prostheses (AREA)
  • Lubricants (AREA)
KR1020217002429A 2013-03-15 2014-03-14 Rna-안내 게놈 편집의 특이성을 증가시키기 위한 rna-안내 foki 뉴클레아제(rfn)의 용도 Active KR102271292B1 (ko)

Applications Claiming Priority (10)

Application Number Priority Date Filing Date Title
US201361799647P 2013-03-15 2013-03-15
US61/799,647 2013-03-15
US201361838178P 2013-06-21 2013-06-21
US201361838148P 2013-06-21 2013-06-21
US61/838,148 2013-06-21
US61/838,178 2013-06-21
US201361921007P 2013-12-26 2013-12-26
US61/921,007 2013-12-26
PCT/US2014/028630 WO2014144288A1 (en) 2013-03-15 2014-03-14 Using rna-guided foki nucleases (rfns) to increase specificity for rna-guided genome editing
KR1020157029177A KR102210322B1 (ko) 2013-03-15 2014-03-14 Rna-안내 게놈 편집의 특이성을 증가시키기 위한 rna-안내 foki 뉴클레아제(rfn)의 용도

Related Parent Applications (1)

Application Number Title Priority Date Filing Date
KR1020157029177A Division KR102210322B1 (ko) 2013-03-15 2014-03-14 Rna-안내 게놈 편집의 특이성을 증가시키기 위한 rna-안내 foki 뉴클레아제(rfn)의 용도

Publications (2)

Publication Number Publication Date
KR20210013304A KR20210013304A (ko) 2021-02-03
KR102271292B1 true KR102271292B1 (ko) 2021-07-02

Family

ID=51537665

Family Applications (8)

Application Number Title Priority Date Filing Date
KR1020157029170A Active KR102210319B1 (ko) 2013-03-15 2014-03-14 특정 게놈 좌위에 대한 유전적 및 후성적 조절 단백질의 rna-안내 표적화
KR1020217002428A Active KR102405549B1 (ko) 2013-03-15 2014-03-14 Rna-안내 게놈 편집을 위해 특이성을 증가시키기 위한 절단된 안내 rna(tru-grnas)의 이용
KR1020217002429A Active KR102271292B1 (ko) 2013-03-15 2014-03-14 Rna-안내 게놈 편집의 특이성을 증가시키기 위한 rna-안내 foki 뉴클레아제(rfn)의 용도
KR1020157029177A Active KR102210322B1 (ko) 2013-03-15 2014-03-14 Rna-안내 게놈 편집의 특이성을 증가시키기 위한 rna-안내 foki 뉴클레아제(rfn)의 용도
KR1020227018265A Active KR102602047B1 (ko) 2013-03-15 2014-03-14 Rna-안내 게놈 편집을 위해 특이성을 증가시키기 위한 절단된 안내 rna(tru-grnas)의 이용
KR1020217002427A Active KR102271291B1 (ko) 2013-03-15 2014-03-14 특정 게놈 좌위에 대한 유전적 및 후성적 조절 단백질의 rna-안내 표적화
KR1020237038728A Active KR102874079B1 (ko) 2013-03-15 2014-03-14 Rna-안내 게놈 편집을 위해 특이성을 증가시키기 위한 절단된 안내 rna(tru-grnas)의 이용
KR1020157029171A Active KR102210323B1 (ko) 2013-03-15 2014-03-14 Rna-안내 게놈 편집을 위해 특이성을 증가시키기 위한 절단된 안내 rna(tru-grnas)의 이용

Family Applications Before (2)

Application Number Title Priority Date Filing Date
KR1020157029170A Active KR102210319B1 (ko) 2013-03-15 2014-03-14 특정 게놈 좌위에 대한 유전적 및 후성적 조절 단백질의 rna-안내 표적화
KR1020217002428A Active KR102405549B1 (ko) 2013-03-15 2014-03-14 Rna-안내 게놈 편집을 위해 특이성을 증가시키기 위한 절단된 안내 rna(tru-grnas)의 이용

Family Applications After (5)

Application Number Title Priority Date Filing Date
KR1020157029177A Active KR102210322B1 (ko) 2013-03-15 2014-03-14 Rna-안내 게놈 편집의 특이성을 증가시키기 위한 rna-안내 foki 뉴클레아제(rfn)의 용도
KR1020227018265A Active KR102602047B1 (ko) 2013-03-15 2014-03-14 Rna-안내 게놈 편집을 위해 특이성을 증가시키기 위한 절단된 안내 rna(tru-grnas)의 이용
KR1020217002427A Active KR102271291B1 (ko) 2013-03-15 2014-03-14 특정 게놈 좌위에 대한 유전적 및 후성적 조절 단백질의 rna-안내 표적화
KR1020237038728A Active KR102874079B1 (ko) 2013-03-15 2014-03-14 Rna-안내 게놈 편집을 위해 특이성을 증가시키기 위한 절단된 안내 rna(tru-grnas)의 이용
KR1020157029171A Active KR102210323B1 (ko) 2013-03-15 2014-03-14 Rna-안내 게놈 편집을 위해 특이성을 증가시키기 위한 절단된 안내 rna(tru-grnas)의 이용

Country Status (12)

Country Link
US (17) US9885033B2 (enExample)
EP (10) EP2971041B1 (enExample)
JP (11) JP6657069B2 (enExample)
KR (8) KR102210319B1 (enExample)
CN (6) CN112301024A (enExample)
AU (10) AU2014239665B2 (enExample)
BR (1) BR112015023489B1 (enExample)
CA (4) CA3161835A1 (enExample)
ES (1) ES2713503T3 (enExample)
IL (2) IL289396B2 (enExample)
WO (4) WO2014152432A2 (enExample)
ZA (1) ZA201506814B (enExample)

Families Citing this family (439)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US8053191B2 (en) 2006-08-31 2011-11-08 Westend Asset Clearinghouse Company, Llc Iterative nucleic acid assembly using activation of vector-encoded traits
EP2625292B1 (en) 2010-10-07 2018-12-05 The General Hospital Corporation Biomarkers of cancer
EP2637780B1 (en) 2010-11-12 2022-02-09 Gen9, Inc. Protein arrays and methods of using and making the same
WO2012078312A2 (en) 2010-11-12 2012-06-14 Gen9, Inc. Methods and devices for nucleic acids synthesis
US10030245B2 (en) 2011-03-23 2018-07-24 E I Du Pont De Nemours And Company Methods for producing a complex transgenic trait locus
CA2841710C (en) 2011-07-15 2021-03-16 The General Hospital Corporation Methods of transcription activator like effector assembly
EP2734621B1 (en) 2011-07-22 2019-09-04 President and Fellows of Harvard College Evaluation and improvement of nuclease cleavage specificity
IL302248A (en) 2011-08-26 2023-06-01 Gen9 Inc Compositions and methods for high fidelity assembly of nucleic acids
EP2766498B1 (en) 2011-10-14 2019-06-19 President and Fellows of Harvard College Sequencing by structure assembly
ES2991004T3 (es) 2011-12-22 2024-12-02 Harvard College Métodos para la detección de analitos
GB201122458D0 (en) 2011-12-30 2012-02-08 Univ Wageningen Modified cascade ribonucleoproteins and uses thereof
WO2013139861A1 (en) 2012-03-20 2013-09-26 Luc Montagnier Methods and pharmaceutical compositions of the treatment of autistic syndrome disorders
US9150853B2 (en) 2012-03-21 2015-10-06 Gen9, Inc. Methods for screening proteins using DNA encoded chemical libraries as templates for enzyme catalysis
AU2013251701A1 (en) 2012-04-24 2014-10-30 Gen9, Inc. Methods for sorting nucleic acids and multiplexed preparative in vitro cloning
EP2841572B1 (en) 2012-04-27 2019-06-19 Duke University Genetic correction of mutated genes
SI3401400T1 (sl) 2012-05-25 2019-10-30 Univ California Postopki in sestavki za RNA usmerjeno modifikacijo tarčne DNA in za RNA usmerjeno modulacijo prepisovanja
US9890364B2 (en) 2012-05-29 2018-02-13 The General Hospital Corporation TAL-Tet1 fusion proteins and methods of use thereof
EP2864531B2 (en) 2012-06-25 2022-08-03 Gen9, Inc. Methods for nucleic acid assembly and high throughput sequencing
EP3789405A1 (en) * 2012-10-12 2021-03-10 The General Hospital Corporation Transcription activator-like effector (tale) - lysine-specific demethylase 1 (lsd1) fusion proteins
EP3138911B1 (en) 2012-12-06 2018-12-05 Sigma Aldrich Co. LLC Crispr-based genome modification and regulation
BR112015013784A2 (pt) 2012-12-12 2017-07-11 Massachusetts Inst Technology aplicação, manipulação e otimização de sistemas, métodos e composições para manipulação de sequência e aplicações terapêuticas
ES2701749T3 (es) 2012-12-12 2019-02-25 Broad Inst Inc Métodos, modelos, sistemas y aparatos para identificar secuencias diana para enzimas Cas o sistemas CRISPR-Cas para secuencias diana y transmitir resultados de los mismos
WO2014093701A1 (en) 2012-12-12 2014-06-19 The Broad Institute, Inc. Functional genomics using crispr-cas systems, compositions, methods, knock out libraries and applications thereof
EP3919505B1 (en) 2013-01-16 2023-08-30 Emory University Uses of cas9-nucleic acid complexes
EP2954042B1 (en) 2013-02-07 2017-12-06 The General Hospital Corporation Tale transcriptional activators
EP3578666A1 (en) 2013-03-12 2019-12-11 President and Fellows of Harvard College Method of generating a three-dimensional nucleic acid containing matrix
US20140315985A1 (en) 2013-03-14 2014-10-23 Caribou Biosciences, Inc. Compositions and methods of nucleic acid-targeting nucleic acids
US10760064B2 (en) 2013-03-15 2020-09-01 The General Hospital Corporation RNA-guided targeting of genetic and epigenomic regulatory proteins to specific genomic loci
US9885033B2 (en) 2013-03-15 2018-02-06 The General Hospital Corporation Increasing specificity for RNA-guided genome editing
US9828582B2 (en) 2013-03-19 2017-11-28 Duke University Compositions and methods for the induction and tuning of gene expression
JP2016522679A (ja) * 2013-04-04 2016-08-04 プレジデント アンド フェローズ オブ ハーバード カレッジ ＣＲＩＳＰＲ／Ｃａｓ系を用いたゲノム編集の治療的使用
SG10201710030QA (en) 2013-06-04 2018-01-30 Harvard College Rna-guided transcriptional regulation
US20140356956A1 (en) * 2013-06-04 2014-12-04 President And Fellows Of Harvard College RNA-Guided Transcriptional Regulation
KR20230136697A (ko) * 2013-06-05 2023-09-26 듀크 유니버시티 Rna-가이드 유전자 편집 및 유전자 조절
JP6625971B2 (ja) 2013-06-17 2019-12-25 ザ・ブロード・インスティテュート・インコーポレイテッド 配列操作のためのタンデムガイド系、方法および組成物の送達、エンジニアリングおよび最適化
EP3011033B1 (en) 2013-06-17 2020-02-19 The Broad Institute, Inc. Functional genomics using crispr-cas systems, compositions methods, screens and applications thereof
EP3011030B1 (en) 2013-06-17 2023-11-08 The Broad Institute, Inc. Optimized crispr-cas double nickase systems, methods and compositions for sequence manipulation
DK3011031T3 (da) 2013-06-17 2020-12-21 Broad Inst Inc Fremføring og anvendelse af crispr-cas-systemerne, vektorer og sammensætninger til levermålretning og -terapi
KR20250012194A (ko) 2013-06-17 2025-01-23 더 브로드 인스티튜트, 인코퍼레이티드 바이러스 구성성분을 사용하여 장애 및 질환을 표적화하기 위한 crispr-cas 시스템 및 조성물의 전달, 용도 및 치료 적용
US10011850B2 (en) 2013-06-21 2018-07-03 The General Hospital Corporation Using RNA-guided FokI Nucleases (RFNs) to increase specificity for RNA-Guided Genome Editing
JP2016528890A (ja) 2013-07-09 2016-09-23 プレジデント アンド フェローズ オブ ハーバード カレッジ ＣＲＩＳＰＲ／Ｃａｓ系を用いるゲノム編集の治療用の使用
WO2015021426A1 (en) * 2013-08-09 2015-02-12 Sage Labs, Inc. A crispr/cas system-based novel fusion protein and its application in genome editing
US20150044192A1 (en) 2013-08-09 2015-02-12 President And Fellows Of Harvard College Methods for identifying a target site of a cas9 nuclease
US9359599B2 (en) 2013-08-22 2016-06-07 President And Fellows Of Harvard College Engineered transcription activator-like effector (TALE) domains and uses thereof
CA3109801C (en) * 2013-08-22 2024-01-09 Andrew Cigan Plant genome modification using guide rna/cas endonuclease systems and methods of use
US9340800B2 (en) 2013-09-06 2016-05-17 President And Fellows Of Harvard College Extended DNA-sensing GRNAS
US9526784B2 (en) 2013-09-06 2016-12-27 President And Fellows Of Harvard College Delivery system for functional nucleases
US9388430B2 (en) 2013-09-06 2016-07-12 President And Fellows Of Harvard College Cas9-recombinase fusion proteins and uses thereof
ES2844174T3 (es) 2013-09-18 2021-07-21 Kymab Ltd Métodos, células y organismos
US10584358B2 (en) 2013-10-30 2020-03-10 North Carolina State University Compositions and methods related to a type-II CRISPR-Cas system in Lactobacillus buchneri
CA2930015A1 (en) 2013-11-07 2015-05-14 Editas Medicine, Inc. Crispr-related methods and compositions with governing grnas
WO2015089364A1 (en) 2013-12-12 2015-06-18 The Broad Institute Inc. Crystal structure of a crispr-cas system, and uses thereof
WO2015089486A2 (en) * 2013-12-12 2015-06-18 The Broad Institute Inc. Systems, methods and compositions for sequence manipulation with optimized functional crispr-cas systems
US9994831B2 (en) * 2013-12-12 2018-06-12 The Regents Of The University Of California Methods and compositions for modifying a single stranded target nucleic acid
KR20160089530A (ko) 2013-12-12 2016-07-27 더 브로드 인스티튜트, 인코퍼레이티드 Ｈｂｖ 및 바이러스 질병 및 질환을 위한 ｃｒｉｓｐｒ­ｃａｓ 시스템 및 조성물의 전달，용도 및 치료적 적용
WO2015089351A1 (en) 2013-12-12 2015-06-18 The Broad Institute Inc. Compositions and methods of use of crispr-cas systems in nucleotide repeat disorders
WO2015089473A1 (en) 2013-12-12 2015-06-18 The Broad Institute Inc. Engineering of systems, methods and optimized guide compositions with new architectures for sequence manipulation
US9840699B2 (en) 2013-12-12 2017-12-12 President And Fellows Of Harvard College Methods for nucleic acid editing
IL289736B2 (en) 2013-12-12 2025-09-01 Massachusetts Inst Technology Administration, use and therapeutic applications of CRISPR–Cas gene editing systems and gene editing preparations
AU2014370416B2 (en) 2013-12-26 2021-03-11 The General Hospital Corporation Multiplex guide RNAs
US10787654B2 (en) 2014-01-24 2020-09-29 North Carolina State University Methods and compositions for sequence guiding Cas9 targeting
PT3105328T (pt) 2014-02-11 2020-07-06 Univ Colorado Regents Engenharia de genomas multiplexada possibilitada por crispr
EP3114227B1 (en) 2014-03-05 2021-07-21 Editas Medicine, Inc. Crispr/cas-related methods and compositions for treating usher syndrome and retinitis pigmentosa
WO2015138510A1 (en) 2014-03-10 2015-09-17 Editas Medicine., Inc. Crispr/cas-related methods and compositions for treating leber's congenital amaurosis 10 (lca10)
US11141493B2 (en) 2014-03-10 2021-10-12 Editas Medicine, Inc. Compositions and methods for treating CEP290-associated disease
US11339437B2 (en) 2014-03-10 2022-05-24 Editas Medicine, Inc. Compositions and methods for treating CEP290-associated disease
WO2015148863A2 (en) 2014-03-26 2015-10-01 Editas Medicine, Inc. Crispr/cas-related methods and compositions for treating sickle cell disease
EP3126495A1 (en) 2014-04-02 2017-02-08 Editas Medicine, Inc. Crispr/cas-related methods and compositions for treating primary open angle glaucoma
JP2017512481A (ja) 2014-04-08 2017-05-25 ノースカロライナ ステート ユニバーシティーＮｏｒｔｈ Ｃａｒｏｌｉｎａ Ｓｔａｔｅ Ｕｎｉｖｅｒｓｉｔｙ Ｃｒｉｓｐｒ関連遺伝子を用いた、ｒｎａ依存性の転写抑制のための方法および組成物
AU2015266770A1 (en) 2014-05-30 2016-12-08 The Board Of Trustees Of The Leland Stanford Junior University Compositions and methods of delivering treatments for latent viral infections
ES2888976T3 (es) 2014-06-23 2022-01-10 Massachusetts Gen Hospital Identificación no sesgada pangenómica de DSBs evaluada por secuenciación (GUIDE-Seq.)
EP3760208B1 (en) 2014-06-25 2024-05-29 The General Hospital Corporation Targeting human satellite ii (hsatii)
US20150376587A1 (en) * 2014-06-25 2015-12-31 Caribou Biosciences, Inc. RNA Modification to Engineer Cas9 Activity
WO2016007604A1 (en) * 2014-07-09 2016-01-14 Gen9, Inc. Compositions and methods for site-directed dna nicking and cleaving
WO2016007839A1 (en) 2014-07-11 2016-01-14 President And Fellows Of Harvard College Methods for high-throughput labelling and detection of biological features in situ using microscopy
CN106687594A (zh) 2014-07-11 2017-05-17 纳幕尔杜邦公司 用于产生对草甘膦除草剂具有抗性的植物的组合物和方法
WO2016022363A2 (en) 2014-07-30 2016-02-11 President And Fellows Of Harvard College Cas9 proteins including ligand-dependent inteins
WO2016022866A1 (en) 2014-08-07 2016-02-11 Agilent Technologies, Inc. Cis-blocked guide rna
EP3633032A3 (en) 2014-08-28 2020-07-29 North Carolina State University Novel cas9 proteins and guiding features for dna targeting and genome editing
WO2016040030A1 (en) 2014-09-12 2016-03-17 E. I. Du Pont De Nemours And Company Generation of site-specific-integration sites for complex trait loci in corn and soybean, and methods of use
WO2016049258A2 (en) * 2014-09-25 2016-03-31 The Broad Institute Inc. Functional screening with optimized functional crispr-cas systems
US20170233762A1 (en) * 2014-09-29 2017-08-17 The Regents Of The University Of California Scaffold rnas
AU2015330699B2 (en) * 2014-10-10 2021-12-02 Editas Medicine, Inc. Compositions and methods for promoting homology directed repair
GB201418965D0 (enExample) 2014-10-24 2014-12-10 Ospedale San Raffaele And Fond Telethon
US9816080B2 (en) 2014-10-31 2017-11-14 President And Fellows Of Harvard College Delivery of CAS9 via ARRDC1-mediated microvesicles (ARMMs)
EP3215623A4 (en) 2014-11-06 2018-09-26 President and Fellows of Harvard College Cells lacking b2m surface expression and methods for allogeneic administration of such cells
EP4464338A3 (en) 2014-11-07 2025-02-12 Editas Medicine, Inc. Systems for improving crispr/cas-mediated genome-editing
KR101828933B1 (ko) * 2014-11-14 2018-02-14 기초과학연구원 유전체에서 유전자 가위의 비표적 위치를 검출하는 방법
US11352666B2 (en) 2014-11-14 2022-06-07 Institute For Basic Science Method for detecting off-target sites of programmable nucleases in a genome
CN104531633A (zh) * 2014-11-18 2015-04-22 李云英 Cas9-scForkI融合蛋白及其应用
EP3222728B1 (en) * 2014-11-19 2021-07-14 Institute for Basic Science Method for regulating gene expression using cas9 protein expressed from two vectors
CN107250148B (zh) 2014-12-03 2021-04-16 安捷伦科技有限公司 具有化学修饰的指导rna
WO2016090385A1 (en) * 2014-12-05 2016-06-09 Applied Stemcell, Inc. Site-directed crispr/recombinase compositions and methods of integrating transgenes
US9888673B2 (en) 2014-12-10 2018-02-13 Regents Of The University Of Minnesota Genetically modified cells, tissues, and organs for treating disease
EP3985115A1 (en) 2014-12-12 2022-04-20 The Broad Institute, Inc. Protected guide rnas (pgrnas)
EP3230452B1 (en) * 2014-12-12 2025-06-11 The Broad Institute, Inc. Dead guides for crispr transcription factors
US20180179523A1 (en) * 2014-12-18 2018-06-28 Integrated Dna Technologies, Inc. Crispr-based compositions and methods of use
CN119320775A (zh) * 2014-12-18 2025-01-17 综合基因技术公司 基于crispr的组合物和使用方法
US10190106B2 (en) * 2014-12-22 2019-01-29 Univesity Of Massachusetts Cas9-DNA targeting unit chimeras
EP3702456A1 (en) 2014-12-24 2020-09-02 The Broad Institute, Inc. Crispr having or associated with destabilization domains
WO2016114972A1 (en) 2015-01-12 2016-07-21 The Regents Of The University Of California Heterodimeric cas9 and methods of use thereof
US11180792B2 (en) 2015-01-28 2021-11-23 The Regents Of The University Of California Methods and compositions for labeling a single-stranded target nucleic acid
SG10201804715WA (en) 2015-01-28 2018-07-30 Pioneer Hi Bred Int Crispr hybrid dna/rna polynucleotides and methods of use
WO2016130600A2 (en) * 2015-02-09 2016-08-18 Duke University Compositions and methods for epigenome editing
HK1248274A1 (zh) * 2015-02-18 2018-10-12 衣阿华州立大学研究基金公司 修饰nf-yc4启动子的转录抑制子结合位点以增加蛋白质含量和抗应力
WO2016141224A1 (en) 2015-03-03 2016-09-09 The General Hospital Corporation Engineered crispr-cas9 nucleases with altered pam specificity
US10450576B2 (en) 2015-03-27 2019-10-22 E I Du Pont De Nemours And Company Soybean U6 small nuclear RNA gene promoters and their use in constitutive expression of small RNA genes in plants
WO2016164356A1 (en) 2015-04-06 2016-10-13 The Board Of Trustees Of The Leland Stanford Junior University Chemically modified guide rnas for crispr/cas-mediated gene regulation
US11180793B2 (en) 2015-04-24 2021-11-23 Editas Medicine, Inc. Evaluation of Cas9 molecule/guide RNA molecule complexes
EP3294342A4 (en) 2015-05-08 2018-11-07 President and Fellows of Harvard College Universal donor stem cells and related methods
JP7030522B2 (ja) 2015-05-11 2022-03-07 エディタス・メディシン、インコーポレイテッド 幹細胞における遺伝子編集のための最適化ｃｒｉｓｐｒ／ｃａｓ９システムおよび方法
US10117911B2 (en) 2015-05-29 2018-11-06 Agenovir Corporation Compositions and methods to treat herpes simplex virus infections
GB2543873A (en) 2015-05-29 2017-05-03 Agenovir Corp Compositions and methods for cell targeted HPV treatment
EP3303607A4 (en) 2015-05-29 2018-10-10 North Carolina State University Methods for screening bacteria, archaea, algae, and yeast using crispr nucleic acids
WO2016196655A1 (en) 2015-06-03 2016-12-08 The Regents Of The University Of California Cas9 variants and methods of use thereof
EP3302525A2 (en) 2015-06-05 2018-04-11 Novartis AG Methods and compositions for diagnosing, treating, and monitoring treatment of shank3 deficiency associated disorders
CN108026526B (zh) 2015-06-09 2023-05-12 爱迪塔斯医药公司 用于改善移植的crispr/cas相关方法和组合物
ES2802524T3 (es) * 2015-06-10 2021-01-20 Firmenich & Cie Líneas celulares para el cribado de receptores de aroma y olor
US20160362705A1 (en) 2015-06-12 2016-12-15 Lonza Walkersville, Inc. Methods for Nuclear Reprogramming Using Synthetic Transcription Factors
DK3307872T3 (da) 2015-06-15 2023-10-23 Univ North Carolina State Fremgangsmåder og sammensætninger til effektiv indgivelse af nukleinsyrer og rna-baserede antimikrober
WO2016205759A1 (en) 2015-06-18 2016-12-22 The Broad Institute Inc. Engineering and optimization of systems, methods, enzymes and guide scaffolds of cas9 orthologs and variants for sequence manipulation
TWI813532B (zh) 2015-06-18 2023-09-01 美商博得學院股份有限公司 降低脱靶效應的ｃｒｉｓｐｒ酶突變
WO2017004279A2 (en) * 2015-06-29 2017-01-05 Massachusetts Institute Of Technology Compositions comprising nucleic acids and methods of using the same
EP3313989B1 (en) * 2015-06-29 2024-12-25 Ionis Pharmaceuticals, Inc. Modified crispr rna and modified single crispr rna and uses thereof
EP3322804B1 (en) 2015-07-15 2021-09-01 Rutgers, The State University of New Jersey Nuclease-independent targeted gene editing platform and uses thereof
WO2017015637A1 (en) 2015-07-22 2017-01-26 Duke University High-throughput screening of regulatory element function with epigenome editing technologies
WO2017023803A1 (en) 2015-07-31 2017-02-09 Regents Of The University Of Minnesota Modified cells and methods of therapy
US20180230450A1 (en) * 2015-08-03 2018-08-16 President And Fellows Of Harvard College Cas9 Genome Editing and Transcriptional Regulation
WO2017024047A1 (en) * 2015-08-03 2017-02-09 Emendobio Inc. Compositions and methods for increasing nuclease induced recombination rate in cells
KR102699944B1 (ko) 2015-08-25 2024-09-13 듀크 유니버시티 Rna-가이드된 엔도뉴클레아제를 이용하는 게놈 조작에서 특이성을 개선하는 조성물 및 방법
KR102668608B1 (ko) * 2015-08-28 2024-05-24 더 제너럴 하스피탈 코포레이션 조작된 crispr-cas9 뉴클레아제
US9512446B1 (en) 2015-08-28 2016-12-06 The General Hospital Corporation Engineered CRISPR-Cas9 nucleases
US9926546B2 (en) 2015-08-28 2018-03-27 The General Hospital Corporation Engineered CRISPR-Cas9 nucleases
CN107922949A (zh) * 2015-08-31 2018-04-17 安捷伦科技有限公司 用于通过同源重组的基于crispr/cas的基因组编辑的化合物和方法
WO2017044776A1 (en) * 2015-09-10 2017-03-16 Texas Tech University System Single-guide rna (sgrna) with improved knockout efficiency
CN108350453A (zh) 2015-09-11 2018-07-31 通用医疗公司 核酸酶dsb的完全查询和测序(find-seq)
AU2016326711B2 (en) 2015-09-24 2022-11-03 Editas Medicine, Inc. Use of exonucleases to improve CRISPR/Cas-mediated genome editing
US11286480B2 (en) 2015-09-28 2022-03-29 North Carolina State University Methods and compositions for sequence specific antimicrobials
CA3000762A1 (en) 2015-09-30 2017-04-06 The General Hospital Corporation Comprehensive in vitro reporting of cleavage events by sequencing (circle-seq)
MX390738B (es) 2015-10-06 2025-03-21 Inst Basic Science Metodo para producir plantas de genoma modificado a partir de protoplastos de planta a alta eficiencia.
WO2017066497A2 (en) 2015-10-13 2017-04-20 Duke University Genome engineering with type i crispr systems in eukaryotic cells
US9677090B2 (en) 2015-10-23 2017-06-13 Caribou Biosciences, Inc. Engineered nucleic-acid targeting nucleic acids
IL258821B (en) 2015-10-23 2022-07-01 Harvard College Nucleobase editors and their uses
CN108474022A (zh) 2015-11-03 2018-08-31 哈佛学院董事及会员团体 用于包含三维核酸的基质容积成像的设备和方法
WO2017081288A1 (en) 2015-11-11 2017-05-18 Lonza Ltd Crispr-associated (cas) proteins with reduced immunogenicity
WO2017087979A1 (en) 2015-11-20 2017-05-26 Washington University Preparative electrophoretic method for targeted purification of genomic dna fragments
WO2017090724A1 (ja) * 2015-11-25 2017-06-01 国立大学法人 群馬大学 Ｄｎａメチル化編集用キットおよびｄｎａメチル化編集方法
AU2016362282B2 (en) 2015-11-30 2023-03-16 Duke University Therapeutic targets for the correction of the human dystrophin gene by gene editing and methods of use
US11345931B2 (en) 2015-12-14 2022-05-31 President And Fellows Of Harvard College Cas discrimination using tuned guide RNA
WO2017112620A1 (en) 2015-12-22 2017-06-29 North Carolina State University Methods and compositions for delivery of crispr based antimicrobials
NZ783532A (en) * 2015-12-28 2025-09-26 Novartis Ag Compositions and methods for the treatment of hemoglobinopathies
EP3901258A1 (en) 2016-01-11 2021-10-27 The Board of Trustees of the Leland Stanford Junior University Chimeric proteins and methods of immunotherapy
NZ743983A (en) 2016-01-11 2025-08-29 Univ Leland Stanford Junior Chimeric proteins and methods of regulating gene expression
WO2017136794A1 (en) 2016-02-03 2017-08-10 Massachusetts Institute Of Technology Structure-guided chemical modification of guide rna and its applications
CN109072205A (zh) 2016-02-10 2018-12-21 密歇根大学董事会 核酸的检测
EP3417061B1 (en) 2016-02-18 2022-10-26 The Regents of the University of California Methods and compositions for gene editing in stem cells
US10538750B2 (en) 2016-02-29 2020-01-21 Agilent Technologies, Inc. Methods and compositions for blocking off-target nucleic acids from cleavage by CRISPR proteins
MX381136B (es) * 2016-03-15 2025-03-12 Rnaissance Ag Llc Métodos y composiciones para incrementar la producción de arn bicatenario.
EP3429567B1 (en) 2016-03-16 2024-01-10 The J. David Gladstone Institutes Methods and compositions for treating obesity and/or diabetes and for identifying candidate treatment agents
EP3219799A1 (en) 2016-03-17 2017-09-20 IMBA-Institut für Molekulare Biotechnologie GmbH Conditional crispr sgrna expression
US11512311B2 (en) 2016-03-25 2022-11-29 Editas Medicine, Inc. Systems and methods for treating alpha 1-antitrypsin (A1AT) deficiency
WO2017165826A1 (en) 2016-03-25 2017-09-28 Editas Medicine, Inc. Genome editing systems comprising repair-modulating enzyme molecules and methods of their use
CN106701765A (zh) * 2016-04-11 2017-05-24 广东赤萌医疗科技有限公司 用于hiv感染治疗的多核苷酸及其制备药物应用
EP4047092B1 (en) 2016-04-13 2025-07-30 Editas Medicine, Inc. Cas9 fusion molecules, gene editing systems, and methods of use thereof
US20190127713A1 (en) * 2016-04-13 2019-05-02 Duke University Crispr/cas9-based repressors for silencing gene targets in vivo and methods of use
CA3022290A1 (en) 2016-04-25 2017-11-02 President And Fellows Of Harvard College Hybridization chain reaction methods for in situ molecular detection
CN107326046A (zh) * 2016-04-28 2017-11-07 上海邦耀生物科技有限公司 一种提高外源基因同源重组效率的方法
MX2018014172A (es) 2016-05-20 2019-08-22 Regeneron Pharma Métodos para romper la tolerancia inmunológica usando múltiples arn guías.
WO2017208247A1 (en) * 2016-06-02 2017-12-07 Yissum Research Development Company Of The Hebrew University Of Jerusalem Ltd. Assay for the removal of methyl-cytosine residues from dna
GB2582731B8 (en) * 2016-06-02 2021-10-27 Sigma Aldrich Co Llc Using programmable DNA binding proteins to enhance targeted genome modification
US10767175B2 (en) 2016-06-08 2020-09-08 Agilent Technologies, Inc. High specificity genome editing using chemically modified guide RNAs
CN109312386B (zh) * 2016-06-15 2022-10-25 株式会社图尔金 使用中靶靶标和脱靶靶标的多重靶标系统筛选靶特异性核酸酶的方法及其用途
WO2017223538A1 (en) 2016-06-24 2017-12-28 The Regents Of The University Of Colorado, A Body Corporate Methods for generating barcoded combinatorial libraries
EP3474849B1 (en) 2016-06-27 2025-05-21 The Broad Institute, Inc. Compositions and methods for detecting and treating diabetes
US11359234B2 (en) 2016-07-01 2022-06-14 Microsoft Technology Licensing, Llc Barcoding sequences for identification of gene expression
EP3478852B1 (en) * 2016-07-01 2020-08-12 Microsoft Technology Licensing, LLC Storage through iterative dna editing
US20180004537A1 (en) 2016-07-01 2018-01-04 Microsoft Technology Licensing, Llc Molecular State Machines
US20230151341A1 (en) * 2016-07-13 2023-05-18 Qihan Chen Method for specifically editing genomic dna and application thereof
EP4219462A1 (en) 2016-07-13 2023-08-02 Vertex Pharmaceuticals Incorporated Methods, compositions and kits for increasing genome editing efficiency
EP4275747A3 (en) 2016-07-19 2024-01-24 Duke University Therapeutic applications of cpf1-based genome editing
CN109790527A (zh) * 2016-07-26 2019-05-21 通用医疗公司 普氏菌属和弗朗西斯氏菌属的CRISPR1(Cpf1)的变体
US11123409B2 (en) * 2016-07-28 2021-09-21 Institute For Basic Science Method of treating or preventing eye disease using Cas9 protein and guide RNA
US10548302B2 (en) 2016-07-29 2020-02-04 Regeneron Pharmaceuticals, Inc. Fibrillin-1 mutations for modeling neonatal progeroid syndrome with congenital lipodystrophy
WO2018026976A1 (en) 2016-08-02 2018-02-08 Editas Medicine, Inc. Compositions and methods for treating cep290 associated disease
KR20250103795A (ko) 2016-08-03 2025-07-07 프레지던트 앤드 펠로우즈 오브 하바드 칼리지 아데노신 핵염기 편집제 및 그의 용도
CA3033327A1 (en) 2016-08-09 2018-02-15 President And Fellows Of Harvard College Programmable cas9-recombinase fusion proteins and uses thereof
WO2018030457A1 (ja) 2016-08-10 2018-02-15 武田薬品工業株式会社 真核細胞のゲノムの標的部位を改変する方法及び標的部位における検出対象核酸配列の存在又は非存在を検出する方法
EP3500675A4 (en) * 2016-08-19 2020-01-29 Whitehead Institute for Biomedical Research Methods of editing dna methylation
JP7050215B2 (ja) 2016-08-19 2022-04-08 ツールゲン インコーポレイテッド 人工的に操作された血管新生調節系
WO2018039438A1 (en) 2016-08-24 2018-03-01 President And Fellows Of Harvard College Incorporation of unnatural amino acids into proteins using base editing
IL264792B2 (en) 2016-08-24 2023-10-01 Sangamo Therapeutics Inc Engineered target-specific zinc-finger nucleases
AU2017315406B2 (en) 2016-08-24 2021-04-01 Sangamo Therapeutics, Inc. Regulation of gene expression using engineered nucleases
WO2018048194A1 (ko) * 2016-09-07 2018-03-15 울산대학교 산학협력단 dCas9 단백질 및 표적 핵산 서열에 결합하는 gRNA를 이용한 핵산 검출의 민감도 및 특이도 향상용 조성물 및 방법
CA3035910A1 (en) * 2016-09-07 2018-03-15 Flagship Pioneering, Inc. Methods and compositions for modulating gene expression
CN110023494A (zh) 2016-09-30 2019-07-16 加利福尼亚大学董事会 Rna指导的核酸修饰酶及其使用方法
CN107880132B (zh) * 2016-09-30 2022-06-17 北京大学 一种融合蛋白及使用其进行同源重组的方法
US10669539B2 (en) 2016-10-06 2020-06-02 Pioneer Biolabs, Llc Methods and compositions for generating CRISPR guide RNA libraries
US11242542B2 (en) 2016-10-07 2022-02-08 Integrated Dna Technologies, Inc. S. pyogenes Cas9 mutant genes and polypeptides encoded by same
KR20230164759A (ko) 2016-10-07 2023-12-04 인티그레이티드 디엔에이 테크놀로지스 아이엔씨. Ｓ. 피오게네스 ｃａｓ９ 돌연변이 유전자 및 이에 의해 암호화되는 폴리펩티드
JP7588390B2 (ja) 2016-10-14 2024-11-22 プレジデント アンド フェローズ オブ ハーバード カレッジ 核酸塩基エディターのａａｖ送達
CN110290813A (zh) 2016-10-14 2019-09-27 通用医疗公司 表观遗传学调控的位点特异性核酸酶
CA3041068A1 (en) 2016-10-18 2018-04-26 Regents Of The University Of Minnesota Tumor infiltrating lymphocytes and methods of therapy
US20180245065A1 (en) 2016-11-01 2018-08-30 Novartis Ag Methods and compositions for enhancing gene editing
US20180282722A1 (en) * 2016-11-21 2018-10-04 Massachusetts Institute Of Technology Chimeric DNA:RNA Guide for High Accuracy Cas9 Genome Editing
JP2019535287A (ja) 2016-11-22 2019-12-12 インテグレイテツド・デイー・エヌ・エイ・テクノロジーズ・インコーポレイテツド Ｃｒｉｓｐｒ／ｃｐｆ１システム及び方法
TWI835719B (zh) * 2016-12-08 2024-03-21 美商英特利亞醫療公司 經修飾之嚮導rna
US20200149039A1 (en) 2016-12-12 2020-05-14 Whitehead Institute For Biomedical Research Regulation of transcription through ctcf loop anchors
US11293022B2 (en) 2016-12-12 2022-04-05 Integrated Dna Technologies, Inc. Genome editing enhancement
CA3045131A1 (en) 2016-12-14 2018-06-21 Ligandal, Inc. Methods and compositions for nucleic acid and protein payload delivery
WO2018119010A1 (en) 2016-12-19 2018-06-28 Editas Medicine, Inc. Assessing nuclease cleavage
KR102551664B1 (ko) * 2016-12-22 2023-07-05 인텔리아 테라퓨틱스, 인크. 알파-1 항트립신 결핍을 치료하기 위한 조성물 및 방법
WO2018119359A1 (en) 2016-12-23 2018-06-28 President And Fellows Of Harvard College Editing of ccr5 receptor gene to protect against hiv infection
EP3562942A4 (en) * 2016-12-28 2020-12-09 Ionis Pharmaceuticals, Inc. MODIFIED CRISPR-RNA AND ITS USES
CN110520163A (zh) 2017-01-05 2019-11-29 新泽西鲁特格斯州立大学 独立于dna双链断裂的靶向基因编辑平台及其用途
US12110545B2 (en) 2017-01-06 2024-10-08 Editas Medicine, Inc. Methods of assessing nuclease cleavage
ES2949801T3 (es) 2017-01-09 2023-10-03 Whitehead Inst Biomedical Res Métodos para alterar la expresión génica mediante la perturbación de multímeros de factores de transcripción que estructuran bucles reguladores
EP3572525A4 (en) * 2017-01-17 2020-09-30 Institute for Basic Science PROCEDURE FOR IDENTIFYING AN OFF-TARGET BASIC EDITING SITE BY BREAKING A SINGLE DNA STRAND
RU2019126483A (ru) 2017-01-23 2021-02-24 Ридженерон Фармасьютикалз, Инк. Варианты 17-бета-гидроксистероиддегидрогеназы 13 (hsd17b13) и их применение
TW201839136A (zh) 2017-02-06 2018-11-01 瑞士商諾華公司 治療血色素異常症之組合物及方法
JP2020507312A (ja) * 2017-02-10 2020-03-12 ザイマージェン インコーポレイテッド 複数の宿主用の複数のｄｎａコンストラクトのアセンブリ及び編集のためのモジュラーユニバーサルプラスミド設計戦略
EP3655533A1 (en) 2017-02-24 2020-05-27 Georg-August-Universität Göttingen Stiftung Öffentlichen Rechts, Universitätsmedizin Method for re-expression of different hypermethylated genes involved in fibrosis, like hypermethylated rasal,1 and use thereof in treatment of fibrosis as well as kit of parts for re-expression of hypermethylated genes including rasal1 in a subject
CN110730826A (zh) 2017-03-08 2020-01-24 密歇根大学董事会 分析物检测
WO2018165504A1 (en) 2017-03-09 2018-09-13 President And Fellows Of Harvard College Suppression of pain by gene editing
CN110662556A (zh) 2017-03-09 2020-01-07 哈佛大学的校长及成员们 癌症疫苗
JP2020510439A (ja) 2017-03-10 2020-04-09 プレジデント アンド フェローズ オブ ハーバード カレッジ シトシンからグアニンへの塩基編集因子
EP3596217A1 (en) 2017-03-14 2020-01-22 Editas Medicine, Inc. Systems and methods for the treatment of hemoglobinopathies
BR112019019655A2 (pt) 2017-03-23 2020-04-22 Harvard College editores de nucleobase que compreendem proteínas de ligação a dna programáveis por ácido nucleico
CN108660161B (zh) * 2017-03-31 2023-05-09 中国科学院脑科学与智能技术卓越创新中心 基于CRISPR/Cas9技术的制备无嵌合基因敲除动物的方法
CN110506203A (zh) * 2017-04-07 2019-11-26 塞奇科学股份有限公司 用于通过使用集成电泳dna纯化来检测遗传结构变异的系统和方法
JP7379160B2 (ja) 2017-04-21 2023-11-14 ザ ジェネラル ホスピタル コーポレイション ＣＲＩＳＰＲ－Ｃｐｆ１を使用する誘導性で調整可能な多重ヒト遺伝子制御
CN110799525A (zh) 2017-04-21 2020-02-14 通用医疗公司 具有改变的PAM特异性的CPF1(CAS12a)的变体
EP3615672A1 (en) 2017-04-28 2020-03-04 Editas Medicine, Inc. Methods and systems for analyzing guide rna molecules
EP3622070A2 (en) 2017-05-10 2020-03-18 Editas Medicine, Inc. Crispr/rna-guided nuclease systems and methods
WO2018209320A1 (en) 2017-05-12 2018-11-15 President And Fellows Of Harvard College Aptazyme-embedded guide rnas for use with crispr-cas9 in genome editing and transcriptional activation
JP7324713B2 (ja) 2017-05-25 2023-08-10 ザ ジェネラル ホスピタル コーポレイション 改善された精度および特異性を有する塩基エディター
CN108977442B (zh) * 2017-06-05 2023-01-06 广州市锐博生物科技有限公司 用于dna编辑的系统及其应用
CN110997906B (zh) 2017-06-05 2024-05-07 雷杰纳荣制药公司 B4galt1变体及其用途
KR102746733B1 (ko) 2017-06-09 2024-12-24 에디타스 메디신, 인코포레이티드 조작된 cas9 뉴클레아제
US10907150B2 (en) 2017-06-14 2021-02-02 Wisconsin Alumni Research Foundation Modified guide RNAs, CRISPR-ribonucleotprotein complexes and methods of use
US20200362355A1 (en) 2017-06-15 2020-11-19 The Regents Of The University Of California Targeted non-viral dna insertions
US9982279B1 (en) 2017-06-23 2018-05-29 Inscripta, Inc. Nucleic acid-guided nucleases
US10011849B1 (en) 2017-06-23 2018-07-03 Inscripta, Inc. Nucleic acid-guided nucleases
JP2020530307A (ja) 2017-06-30 2020-10-22 インティマ・バイオサイエンス，インコーポレーテッド 遺伝子治療のためのアデノ随伴ウイルスベクター
WO2019003193A1 (en) 2017-06-30 2019-01-03 Novartis Ag METHODS FOR TREATING DISEASES USING GENE EDITING SYSTEMS
KR102523217B1 (ko) * 2017-07-11 2023-04-20 시그마-알드리치 컴퍼니., 엘엘씨 표적된 게놈 변형을 개선하기 위한 뉴클레오솜 상호작용 단백질 도메인 사용
EP3652312A1 (en) 2017-07-14 2020-05-20 Editas Medicine, Inc. Systems and methods for targeted integration and genome editing and detection thereof using integrated priming sites
JP2020534795A (ja) 2017-07-28 2020-12-03 プレジデント アンド フェローズ オブ ハーバード カレッジ ファージによって支援される連続的進化（ｐａｃｅ）を用いて塩基編集因子を進化させるための方法および組成物
WO2019028032A1 (en) 2017-07-31 2019-02-07 Regeneron Pharmaceuticals, Inc. EMBRYONIC STEM CELLS OF TRANSGENIC MOUSE CASES AND MICE AND USES THEREOF
JP2020533957A (ja) * 2017-07-31 2020-11-26 リジェネロン・ファーマシューティカルズ・インコーポレイテッドＲｅｇｅｎｅｒｏｎ Ｐｈａｒｍａｃｅｕｔｉｃａｌｓ， Ｉｎｃ． Ｃｒｉｓｐｒリポーター非ヒト動物およびその使用
WO2019028029A1 (en) 2017-07-31 2019-02-07 Regeneron Pharmaceuticals, Inc. EVALUATION OF CRISPR / CAS INDUCED RECOMBINATION WITH IN VIVO EXOGENIC DONOR NUCLEIC ACID
CN111278848B (zh) 2017-08-04 2023-06-27 北京大学 特异性识别甲基化修饰dna碱基的tale rvd及其应用
CN111278983A (zh) 2017-08-08 2020-06-12 北京大学 基因敲除方法
WO2019040650A1 (en) 2017-08-23 2019-02-28 The General Hospital Corporation GENETICALLY MODIFIED CRISPR-CAS9 NUCLEASES HAVING MODIFIED PAM SPECIFICITY
US11319532B2 (en) 2017-08-30 2022-05-03 President And Fellows Of Harvard College High efficiency base editors comprising Gam
EP3585162B1 (en) 2017-09-29 2023-08-30 Regeneron Pharmaceuticals, Inc. Rodents comprising a humanized ttr locus and methods of use
EP3694993A4 (en) 2017-10-11 2021-10-13 The General Hospital Corporation SITE-SPECIFIC AND PARASITIC GENOMIC DESAMINATION DETECTION METHODS INDUCED BY BASIC EDITING TECHNOLOGIES
KR20250107288A (ko) 2017-10-16 2025-07-11 더 브로드 인스티튜트, 인코퍼레이티드 아데노신 염기 편집제의 용도
CN107602707B (zh) * 2017-10-17 2021-04-23 湖北大学 一种特异性调节枯草芽孢杆菌外源基因表达的dcas9-ω融合蛋白及其应用
IL274179B2 (en) 2017-10-27 2024-02-01 Univ California Targeted replacement of endogenous T cell receptors
KR20200075000A (ko) * 2017-11-01 2020-06-25 에디타스 메디신, 인코포레이티드 면역요법을 위한 t 세포 내 tgfbr2의 crispr-cas9 편집 방법, 조성물 및 성분
WO2019087113A1 (en) 2017-11-01 2019-05-09 Novartis Ag Synthetic rnas and methods of use
JP7423520B2 (ja) * 2017-11-16 2024-01-29 アストラゼネカ・アクチエボラーグ Ｃａｓ９ベースノックイン方針の効力を改善するための組成物及び方法
WO2019118949A1 (en) 2017-12-15 2019-06-20 The Broad Institute, Inc. Systems and methods for predicting repair outcomes in genetic engineering
CN111684069A (zh) * 2017-12-22 2020-09-18 G+Flas生命科学有限公司 嵌合基因组工程分子和方法
CN109504711A (zh) * 2018-02-14 2019-03-22 复旦大学 基于CRISPR/cas9和过氧化物酶APEX2系统识别分析特异性基因组位点相互作用DNA的方法
WO2019161340A1 (en) 2018-02-19 2019-08-22 Yale University Phosphopeptide-encoding oligonucleotide libraries and methods for detecting phosphorylation-dependent molecular interactions
US12084676B2 (en) 2018-02-23 2024-09-10 Pioneer Hi-Bred International, Inc. Cas9 orthologs
CA3093702A1 (en) 2018-03-14 2019-09-19 Editas Medicine, Inc. Systems and methods for the treatment of hemoglobinopathies
CN116349651A (zh) 2018-03-19 2023-06-30 瑞泽恩制药公司 使用crispr/cas系统对动物进行转录调制
WO2019195738A1 (en) 2018-04-06 2019-10-10 Children's Medical Center Corporation Compositions and methods for somatic cell reprogramming and modulating imprinting
WO2019204378A1 (en) 2018-04-17 2019-10-24 The General Hospital Corporation Sensitive in vitro assays for substrate preferences and sites of nucleic acid binding, modifying, and cleaving agents
CN117534769A (zh) 2018-04-19 2024-02-09 加利福尼亚大学董事会 用于基因编辑的组合物和方法
WO2019213430A1 (en) * 2018-05-03 2019-11-07 The Board Of Trustees Of The Leland Stanford Junior University Compositions and methods for nicking target dna sequences
CN108588123A (zh) * 2018-05-07 2018-09-28 南京医科大学 CRISPR/Cas9载体组合在制备基因敲除猪的血液制品中的应用
JP7642531B2 (ja) 2018-05-11 2025-03-10 ビーム セラピューティクス インク． プログラム可能塩基エディターシステムを用いて病原性アミノ酸を置換する方法
KR20210045360A (ko) 2018-05-16 2021-04-26 신테고 코포레이션 가이드 rna 설계 및 사용을 위한 방법 및 시스템
WO2019226953A1 (en) 2018-05-23 2019-11-28 The Broad Institute, Inc. Base editors and uses thereof
EP3575396A1 (en) * 2018-06-01 2019-12-04 Algentech SAS Gene targeting
US12227776B2 (en) 2018-06-13 2025-02-18 Caribou Biosciences, Inc. Engineered cascade components and cascade complexes
CN110592141B (zh) * 2018-06-13 2023-07-07 中国科学院上海有机化学研究所 用于调控基因编辑效率的化合物及其应用
US10227576B1 (en) 2018-06-13 2019-03-12 Caribou Biosciences, Inc. Engineered cascade components and cascade complexes
AU2019291918B2 (en) 2018-06-29 2025-06-12 Editas Medicine, Inc. Synthetic guide molecules, compositions and methods relating thereto
CN112513270B (zh) 2018-07-13 2025-02-25 加利福尼亚大学董事会 基于逆转录转座子的递送媒介物及其使用方法
CN112703250A (zh) 2018-08-15 2021-04-23 齐默尔根公司 CRISPRi在高通量代谢工程中的应用
US12275964B2 (en) 2018-08-22 2025-04-15 The Regents Of The University Of California Variant type V CRISPR/Cas effector polypeptides and methods of use thereof
US11834686B2 (en) 2018-08-23 2023-12-05 Sangamo Therapeutics, Inc. Engineered target specific base editors
CN112654702B (zh) 2018-09-07 2025-05-13 阿斯利康(瑞典)有限公司 改进的核酸酶的组合物和方法
US12264330B2 (en) 2018-10-01 2025-04-01 North Carolina State University Recombinant type I CRISPR-Cas system and uses thereof for killing target cells
US12203123B2 (en) 2018-10-01 2025-01-21 North Carolina State University Recombinant type I CRISPR-Cas system and uses thereof for screening for variant cells
US12264313B2 (en) 2018-10-01 2025-04-01 North Carolina State University Recombinant type I CRISPR-Cas system and uses thereof for genome modification and alteration of expression
EP3861120A4 (en) 2018-10-01 2023-08-16 North Carolina State University Recombinant type i crispr-cas system
WO2020076976A1 (en) 2018-10-10 2020-04-16 Readcoor, Inc. Three-dimensional spatial molecular indexing
US11407995B1 (en) 2018-10-26 2022-08-09 Inari Agriculture Technology, Inc. RNA-guided nucleases and DNA binding proteins
WO2020092453A1 (en) 2018-10-29 2020-05-07 The Broad Institute, Inc. Nucleobase editors comprising geocas9 and uses thereof
US11434477B1 (en) 2018-11-02 2022-09-06 Inari Agriculture Technology, Inc. RNA-guided nucleases and DNA binding proteins
US11739320B2 (en) 2018-11-05 2023-08-29 Wisconsin Alumni Research Foundation Gene correction of Pompe disease and other autosomal recessive disorders via RNA-guided nucleases
EP3877517A4 (en) 2018-11-09 2022-09-07 Inari Agriculture, Inc. RNA-DRIVEN NUCLEASES AND DNA-BINDING PROTEINS
WO2020123887A2 (en) 2018-12-14 2020-06-18 Pioneer Hi-Bred International, Inc. Novel crispr-cas systems for genome editing
CN113423831B (zh) 2018-12-20 2023-03-10 瑞泽恩制药公司 核酸酶介导的重复扩增
WO2020148206A1 (en) 2019-01-14 2020-07-23 INSERM (Institut National de la Santé et de la Recherche Médicale) Methods and kits for generating and selecting a variant of a binding protein with increased binding affinity and/or specificity
US12351837B2 (en) 2019-01-23 2025-07-08 The Broad Institute, Inc. Supernegatively charged proteins and uses thereof
WO2020163396A1 (en) 2019-02-04 2020-08-13 The General Hospital Corporation Adenine dna base editor variants with reduced off-target rna editing
WO2020163856A1 (en) 2019-02-10 2020-08-13 The J. David Gladstone Institutes, A Testamentary Trust Established Under The Will Of J. David Gladstone Modified mitochondrion and methods of use thereof
CN113811607A (zh) 2019-03-07 2021-12-17 加利福尼亚大学董事会 CRISPR-Cas效应子多肽和其使用方法
US11781131B2 (en) 2019-03-18 2023-10-10 Regeneron Pharmaceuticals, Inc. CRISPR/Cas dropout screening platform to reveal genetic vulnerabilities associated with tau aggregation
WO2020190932A1 (en) 2019-03-18 2020-09-24 Regeneron Pharmaceuticals, Inc. Crispr/cas screening platform to identify genetic modifiers of tau seeding or aggregation
EP3942042A1 (en) 2019-03-19 2022-01-26 The Broad Institute, Inc. Methods and compositions for editing nucleotide sequences
PH12021500032A1 (en) 2019-04-03 2022-05-02 Regeneron Pharma Methods and compositions for insertion of antibody coding sequences into a safe harbor locus
IL286917B (en) 2019-04-04 2022-09-01 Regeneron Pharma Methods for scarless introduction of targeted modifications into targeting vectors
CN117178959A (zh) 2019-04-04 2023-12-08 瑞泽恩制药公司 包括人源化凝血因子12基因座的非人动物
CN120041438A (zh) 2019-04-12 2025-05-27 阿斯利康(瑞典)有限公司 用于改进的基因编辑的组合物和方法
EP3956349A1 (en) 2019-04-17 2022-02-23 The Broad Institute, Inc. Adenine base editors with reduced off-target effects
AU2020286382A1 (en) 2019-06-04 2021-11-04 Regeneron Pharmaceuticals, Inc. Non-human animals comprising a humanized TTR locus with a beta-slip mutation and methods of use
MX2021015122A (es) 2019-06-07 2022-04-06 Regeneron Pharma Animales no humanos que comprenden un locus de albumina humanizado.
ES3034102T3 (en) 2019-06-14 2025-08-13 Regeneron Pharma Models of tauopathy
EP3783104A1 (en) * 2019-08-20 2021-02-24 Kemijski Institut Coiled-coil mediated tethering of crispr-cas and exonucleases for enhanced genome editing
CA3153980A1 (en) 2019-09-13 2021-03-18 Regeneron Pharmaceuticals, Inc. Transcription modulation in animals using crispr/cas systems delivered by lipid nanoparticles
JP2022548399A (ja) 2019-09-23 2022-11-18 オメガ セラピューティクス， インコーポレイテッド 肝細胞核因子４－アルファ（ＨＮＦ４α）遺伝子発現をモジュレートするための組成物および方法
US12435330B2 (en) 2019-10-10 2025-10-07 The Broad Institute, Inc. Methods and compositions for prime editing RNA
EP3812472B1 (en) 2019-10-21 2022-11-23 Albert-Ludwigs-Universität Freiburg A truly unbiased in vitro assay to profile off-target activity of one or more target-specific programmable nucleases in cells (abnoba-seq)
US11331333B2 (en) 2019-11-08 2022-05-17 Georg-August-Universität Göttingen Stiftung Öffentichen Rechts, Universitätsmadizin Treatment of aberrant fibroblast proliferation
IL292605B2 (en) 2019-11-08 2025-09-01 Regeneron Pharma CRISPR and AAV strategies for treating childhood X-linked retinoschisis
WO2021108363A1 (en) 2019-11-25 2021-06-03 Regeneron Pharmaceuticals, Inc. Crispr/cas-mediated upregulation of humanized ttr allele
US20230042198A1 (en) * 2019-11-25 2023-02-09 La Jolla Institute For Immunology Methods and Compositions for Modulationg Heterochromatin Dysfunction, Genomic Instability, and Associate Conditions
EP4073249A1 (en) 2019-12-11 2022-10-19 Intellia Therapeutics, Inc. Modified guide rnas for gene editing
CN111088357B (zh) * 2019-12-31 2022-09-20 深圳大学 针对escc的肿瘤标志物及其应用
US12312613B2 (en) 2020-01-24 2025-05-27 The General Hospital Corporation Unconstrained genome targeting with near-PAMless engineered CRISPR-Cas9 variants
US12264341B2 (en) 2020-01-24 2025-04-01 The General Hospital Corporation CRISPR-Cas enzymes with enhanced on-target activity
AU2021219795A1 (en) 2020-02-12 2022-08-25 Massachusetts Eye And Ear Infirmary Haplotype-based treatment of RP1 associated retinal degenerations
CN115485385A (zh) 2020-03-04 2022-12-16 瑞泽恩制药公司 用于使肿瘤细胞对免疫疗法敏感的方法和组合物
US20230122226A1 (en) * 2020-03-05 2023-04-20 Board Of Regents Of The University Of Nebraska Crispr/cas9 system for multistrain hiv-1 treatment
EP4125348A1 (en) 2020-03-23 2023-02-08 Regeneron Pharmaceuticals, Inc. Non-human animals comprising a humanized ttr locus comprising a v30m mutation and methods of use
GB2632565B (en) * 2020-04-09 2025-06-04 Verve Therapeutics Inc Base editing of PCSK9 and methods of using same for treatment of disease
AU2021267334A1 (en) * 2020-05-04 2022-12-22 Bluerock Therapeutics Lp Selection by essential-gene knock-in
EP4146797A1 (en) 2020-05-06 2023-03-15 Orchard Therapeutics (Europe) Limited Treatment for neurodegenerative diseases
WO2021226558A1 (en) 2020-05-08 2021-11-11 The Broad Institute, Inc. Methods and compositions for simultaneous editing of both strands of a target double-stranded nucleotide sequence
JP2023526007A (ja) 2020-05-13 2023-06-20 アンスティチュ ナショナル ドゥ ラ サンテ エ ドゥ ラ ルシェルシュ メディカル β－ヘモグロビン異常症の処置のための塩基編集アプローチ
WO2021246165A1 (ja) * 2020-06-03 2021-12-09 国立大学法人広島大学 Ｏａｓｉｓ遺伝子の脱メチル化のための核酸及びそれを用いた脱メチル化方法
GB2612466A (en) * 2020-06-05 2023-05-03 Univ California Compositions and methods for epigenome editing
US20230235315A1 (en) 2020-07-10 2023-07-27 Horizon Discovery Limited Method for producing genetically modified cells
KR20230051223A (ko) 2020-08-11 2023-04-17 이슘 리서치 디벨롭먼트 컴퍼니 오브 더 히브루 유니버시티 오브 예루살렘, 엘티디. Wwox 연관 질병의 치료 방법
US20220049303A1 (en) 2020-08-17 2022-02-17 Readcoor, Llc Methods and systems for spatial mapping of genetic variants
KR102674574B1 (ko) * 2020-09-02 2024-06-13 한국과학기술연구원 Cas9을 위한 신규 tracrRNA 시스템
KR20230082676A (ko) 2020-10-13 2023-06-08 쌍트르 나시오날 드 라 르쉐르쉐 싸이엉띠피끄(쎄.엔.에르.에스.) 표적-항균-플라스미드 조합 접합 및 crispr/cas 시스템 및 그의 용도
CN112430622A (zh) * 2020-10-26 2021-03-02 扬州大学 一种FokI和dCpf1融合蛋白表达载体及其介导的定点基因编辑方法
RU2762831C1 (ru) * 2020-10-26 2021-12-23 Федеральное государственное бюджетное научное учреждение "Всероссийский научно-исследовательский институт сельскохозяйственной биотехнологии" (ФГБНУ ВНИИСБ) Молекула рнк-проводника для геномного редактирования протомоторной области гена vrn-a1 однодольных зерновых с применением системы crispr/cas9
WO2022120022A1 (en) 2020-12-02 2022-06-09 Regeneron Pharmaceuticals, Inc. Crispr sam biosensor cell lines and methods of use thereof
KR20220082186A (ko) 2020-12-10 2022-06-17 한세준 유,무선 충전이 가능한 보조배터리형 uv-led살균기
CA3207144A1 (en) 2021-01-05 2022-07-14 Horizon Discovery Limited Method for producing genetically modified cells
US12171813B2 (en) 2021-02-05 2024-12-24 Christiana Care Gene Editing Institute, Inc. Methods of and compositions for reducing gene expression and/or activity
US20240052372A1 (en) 2021-02-25 2024-02-15 INSERM (Institut National de la Santé et de la Recherche Médicale) Allele-specific genome editing of the nr2e3 mutation g56r
KR102882704B1 (ko) 2021-03-03 2025-11-12 중앙대학교 산학협력단 CRISPR/Cas9 시스템을 이용한 유전체 단일염기 편집 방법 및 이의 용도
CN113846019B (zh) * 2021-03-05 2023-08-01 海南师范大学 一种海洋微拟球藻靶向表观基因组遗传调控方法
EP4095243A1 (en) 2021-05-25 2022-11-30 European Molecular Biology Laboratory System for hybridization-based precision genome cleavage and editing, and uses thereof
WO2022248645A1 (en) 2021-05-27 2022-12-01 Astrazeneca Ab Cas9 effector proteins with enhanced stability
EP4352225A1 (en) 2021-06-10 2024-04-17 Intellia Therapeutics, Inc. Modified guide rnas comprising an internal linker for gene editing
EP4377459A2 (en) 2021-07-30 2024-06-05 Tune Therapeutics, Inc. Compositions and methods for modulating expression of frataxin (fxn)
WO2023010135A1 (en) 2021-07-30 2023-02-02 Tune Therapeutics, Inc. Compositions and methods for modulating expression of methyl-cpg binding protein 2 (mecp2)
CA3229450A1 (en) 2021-08-20 2023-02-23 Wisconsin Alumni Research Foundation Nonviral generation of genome edited chimeric antigen receptor t cells
US11884915B2 (en) 2021-09-10 2024-01-30 Agilent Technologies, Inc. Guide RNAs with chemical modification for prime editing
WO2023056291A1 (en) 2021-09-28 2023-04-06 Acrigen Biosciences Compositions and methods for nucleic acid modifications
WO2023052366A1 (en) 2021-09-28 2023-04-06 INSERM (Institut National de la Santé et de la Recherche Médicale) Base editing approaches for the treatment of beta-hemoglobinopathies
EP4408996A2 (en) 2021-09-30 2024-08-07 Astrazeneca AB Use of inhibitors to increase efficiency of crispr/cas insertions
EP4416292A2 (en) 2021-10-14 2024-08-21 Arsenal Biosciences, Inc. Immune cells having co-expressed shrnas and logic gate systems
WO2023069987A1 (en) 2021-10-20 2023-04-27 University Of Rochester Rejuvenation treatment of age-related white matter loss cross reference to related application
CN118251491A (zh) 2021-10-28 2024-06-25 瑞泽恩制药公司 用于敲除C5的CRISPR/Cas相关方法及组合物
WO2023081689A2 (en) 2021-11-03 2023-05-11 Intellia Therapeutics, Inc. Polynucleotides, compositions, and methods for genome editing
CA3237482A1 (en) 2021-11-03 2023-05-11 The J. David Gladstone Institutes, A Testamentary Trust Established Under The Will Of J. David Gladstone Precise genome editing using retrons
WO2023099591A1 (en) 2021-12-01 2023-06-08 INSERM (Institut National de la Santé et de la Recherche Médicale) Methods for increasing fetal hemoglobin content by editing the +55-kb region of the erythroid-specific bcl11a enhancer
CN118632622A (zh) 2021-12-08 2024-09-10 瑞泽恩制药公司 突变型肌纤蛋白疾病模型及其用途
US20230279442A1 (en) 2021-12-15 2023-09-07 Versitech Limited Engineered cas9-nucleases and method of use thereof
CA3247927A1 (en) 2022-01-14 2023-07-20 Tune Therapeutics, Inc. Compositions, systems and methods for programming T-lymphocyte phenotypes by targeted gene repression
CA3247928A1 (en) 2022-01-14 2023-07-20 Tune Therapeutics, Inc. Compositions, systems and methods for programming T-lymphocyte phenotypes by targeted gene repression
WO2023141487A1 (en) * 2022-01-20 2023-07-27 Inari Agriculture Technology, Inc. Improved soybean explant preparation and transformation
WO2023141602A2 (en) 2022-01-21 2023-07-27 Renagade Therapeutics Management Inc. Engineered retrons and methods of use
US20250161492A1 (en) 2022-01-25 2025-05-22 Institut National de la Santé et de la Recherche Médicale Base editing approaches for the treatment of beta-thalassemia
EP4473103A2 (en) 2022-02-02 2024-12-11 Regeneron Pharmaceuticals, Inc. Anti-tfr:gaa and anti-cd63:gaa insertions for treatment of pompe disease
WO2023152351A1 (en) 2022-02-14 2023-08-17 INSERM (Institut National de la Santé et de la Recherche Médicale) Treatment of liver cancers by disrupting the beta-catenin/tcf-4 binding site located upstream of meg3 in the dlk1/dio3 locus
JP2025514304A (ja) 2022-04-29 2025-05-02 リジェネロン・ファーマシューティカルズ・インコーポレイテッド 遺伝子治療法のための組織特異的遺伝子外セーフハーバーの同定
CA3256953A1 (en) 2022-05-09 2023-11-16 Regeneron Pharmaceuticals, Inc. VECTORS AND METHODS FOR IN VIVO ANTIBODY PRODUCTION
WO2023217888A1 (en) 2022-05-10 2023-11-16 Institut National de la Santé et de la Recherche Médicale Base editing approaches for correcting the cd39 (cag>tag) mutation in patients suffering from βeta-thalassemia
WO2023235725A2 (en) 2022-05-31 2023-12-07 Regeneron Pharmaceuticals, Inc. Crispr-based therapeutics for c9orf72 repeat expansion disease
JP2025521154A (ja) 2022-05-31 2025-07-08 リジェネロン・ファーマシューティカルズ・インコーポレイテッド Ｃ９ｏｒｆ７２反復伸長疾患のためのｃｒｉｓｐｒ干渉療法
CN119731321A (zh) 2022-06-24 2025-03-28 图恩疗法股份有限公司 通过靶向基因阻遏减少低密度脂蛋白的组合物、系统和方法
EP4554967A2 (en) 2022-07-12 2025-05-21 Tune Therapeutics, Inc. Compositions, systems, and methods for targeted transcriptional activation
CN120344660A (zh) 2022-07-18 2025-07-18 雷纳嘉德医疗管理公司 基因编辑组分、系统和使用方法
WO2024018056A1 (en) 2022-07-22 2024-01-25 Institut National de la Santé et de la Recherche Médicale Base editing approaches for correcting the ivs2-1 (g>a) mutation in patients suffering from βeta-thalassemia
CN120659627A (zh) 2022-07-29 2025-09-16 瑞泽恩制药公司 用于转铁蛋白受体（tfr）介导的脑和肌肉递送的组合物和方法
JP2025527567A (ja) 2022-08-19 2025-08-22 チューン セラピューティクス インコーポレイテッド ターゲティングされた遺伝子抑制によるｂ型肝炎ウイルスの調節のための組成物、システム、および方法
WO2024044723A1 (en) 2022-08-25 2024-02-29 Renagade Therapeutics Management Inc. Engineered retrons and methods of use
WO2024047247A1 (en) 2022-09-02 2024-03-07 Institut National de la Santé et de la Recherche Médicale Base editing approaches for the treatment of amyotrophic lateral sclerosis
CA3268005A1 (en) 2022-09-19 2024-03-28 Tune Therapeutics, Inc. Compositions, systems and methods of T lymphocyte function modulation
CN120265314A (zh) 2022-09-28 2025-07-04 瑞泽恩制药公司 抗体抗性修饰受体以增强基于细胞的疗法
JP2025534904A (ja) 2022-10-21 2025-10-21 キージーン ナムローゼ フェンノートシャップ 修飾ｒｎａによる植物細胞へのｒｎａトランスフェクション
US20240182561A1 (en) 2022-11-04 2024-06-06 Regeneron Pharmaceuticals, Inc. Calcium voltage-gated channel auxiliary subunit gamma 1 (cacng1) binding proteins and cacng1-mediated delivery to skeletal muscle
JP2025538220A (ja) 2022-11-14 2025-11-26 リジェネロン・ファーマシューティカルズ・インコーポレイテッド アストロサイトへの線維芽細胞増殖因子受容体３媒介送達のための組成物および方法
CN115820603B (zh) * 2022-11-15 2024-07-05 吉林大学 一种基于dCasRx-NSUN6单基因特异性M5C修饰编辑方法
WO2024121354A1 (en) 2022-12-08 2024-06-13 Keygene N.V. Duplex sequencing with covalently closed dna ends
WO2024131940A1 (zh) * 2022-12-23 2024-06-27 益杰立科(上海)生物科技有限公司 融合物及其用途
WO2024163678A2 (en) 2023-02-01 2024-08-08 Tune Therapeutics, Inc. Fusion proteins and systems for targeted activation of frataxin (fxn) and related methods
WO2024163683A2 (en) 2023-02-01 2024-08-08 Tune Therapeutics, Inc. Systems, compositions, and methods for modulating expression of methyl-cpg binding protein 2 (mecp2) and x-inactive specific transcript (xist)
WO2024165484A1 (en) 2023-02-06 2024-08-15 Institut National de la Santé et de la Recherche Médicale Enrichment of genetically modified hematopoietic stem cells through multiplex base editing
CN116376975B (zh) * 2023-02-27 2024-05-14 中国科学院脑科学与智能技术卓越创新中心 激活异染色质基因的方法及应用
WO2024186890A1 (en) 2023-03-06 2024-09-12 Intellia Therapeutics, Inc. Compositions and methods for hepatitis b virus (hbv) genome editing
CN118684781A (zh) * 2023-03-21 2024-09-24 深圳赫兹生命科学技术有限公司 GnRH-VLP重组去势疫苗及其制备方法
WO2024201368A1 (en) 2023-03-29 2024-10-03 Astrazeneca Ab Use of inhibitors to increase efficiency of crispr/cas insertions
WO2024209000A1 (en) 2023-04-04 2024-10-10 Keygene N.V. Linkers for duplex sequencing
AU2024270764A1 (en) 2023-05-15 2025-12-04 Nchroma Bio, Inc. Compositions and methods for epigenetic regulation of hbv gene expression
US20250002946A1 (en) 2023-06-30 2025-01-02 Regeneron Pharmaceuticals, Inc. Methods And Compositions For Increasing Homology-Directed Repair
WO2025017030A1 (en) 2023-07-17 2025-01-23 Institut National de la Santé et de la Recherche Médicale Prime editing of the -200 region in the hbg1 and/or hbg2 promoter for increasing fetal hemoglobin content in a eukaryotic cell
WO2025017033A1 (en) 2023-07-17 2025-01-23 Institut National de la Santé et de la Recherche Médicale Prime editing of the -115 region in the hbg1 and/or hbg2 promoter for increasing fetal hemoglobin content in a eukaryotic cell
KR20250016657A (ko) * 2023-07-21 2025-02-04 한국화학연구원 dxCas9 및 CRP 유도체를 포함하는, 표적 유전자 발현 조절 시스템 및 이의 제조방법
WO2025029654A2 (en) 2023-07-28 2025-02-06 Regeneron Pharmaceuticals, Inc. Use of bgh-sv40l tandem polya to enhance transgene expression during unidirectional gene insertion
WO2025029657A2 (en) 2023-07-28 2025-02-06 Regeneron Pharmaceuticals, Inc. Anti-tfr:gaa and anti-cd63:gaa insertion for treatment of pompe disease
US20250049896A1 (en) 2023-07-28 2025-02-13 Regeneron Pharmaceuticals, Inc. Anti-tfr:acid sphingomyelinase for treatment of acid sphingomyelinase deficiency
WO2025029835A1 (en) 2023-07-31 2025-02-06 Tune Therapeutics, Inc. Compositions and methods for modulating il-2 gene expression
WO2025029840A1 (en) 2023-07-31 2025-02-06 Tune Therapeutics, Inc. Compositions and methods for multiplexed activation and repression of t cell gene expression
WO2025038494A1 (en) 2023-08-11 2025-02-20 Tune Therapeutics, Inc. Compositions, systems, and methods for lymphoid cell differentiation using targeted gene activation
TW202515994A (zh) 2023-08-14 2025-04-16 美商英特利亞醫療公司 用於對ｃｄ７０進行基因修飾之組合物及方法
TW202521564A (zh) 2023-08-14 2025-06-01 美商英特利亞醫療公司 用於基於細胞之療法的ｃｄ７０ ｃａｒ－ｔ組合物及方法
WO2025038637A1 (en) 2023-08-14 2025-02-20 Intellia Therapeutics, Inc. Compositions and methods for genetically modifying transforming growth factor beta receptor type 2 (tgfβr2)
TW202515992A (zh) 2023-08-14 2025-04-16 美商英特利亞醫療公司 用於對轉形生長因子β受體２型（ＴＧＦβＲ２）進行基因修飾之組合物及方法
WO2025049524A1 (en) 2023-08-28 2025-03-06 Regeneron Pharmaceuticals, Inc. Cxcr4 antibody-resistant modified receptors
WO2025049959A2 (en) 2023-09-01 2025-03-06 Renagade Therapeutics Management Inc. Gene editing systems, compositions, and methods for treatment of vexas syndrome
WO2025059073A1 (en) 2023-09-11 2025-03-20 Tune Therapeutics, Inc. Epigenetic editing methods and systems for differentiating stem cells
WO2025064408A1 (en) 2023-09-18 2025-03-27 The Broad Institute, Inc. Compositions and methods for treating cardiovascular disease
WO2025081042A1 (en) 2023-10-12 2025-04-17 Renagade Therapeutics Management Inc. Nickase-retron template-based precision editing system and methods of use
WO2025090427A1 (en) 2023-10-23 2025-05-01 University Of Rochester Glial-targeted relief of hyperexcitability in neurodegenerative diseases
WO2025096638A2 (en) 2023-10-30 2025-05-08 Turnstone Biologics Corp. Genetically modified tumor infilitrating lymphocytes and methods of producing and using the same
WO2025117544A1 (en) 2023-11-29 2025-06-05 The Broad Institute, Inc. Engineered omega guide molecule and iscb compositions, systems, and methods of use thereof
WO2025155753A2 (en) 2024-01-17 2025-07-24 Renagade Therapeutics Management Inc. Improved gene editing system, guides, and methods
WO2025174765A1 (en) 2024-02-12 2025-08-21 Renagade Therapeutics Management Inc. Lipid nanoparticles comprising coding rna molecules for use in gene editing and as vaccines and therapeutic agents
WO2025184567A1 (en) 2024-03-01 2025-09-04 Regeneron Pharmaceuticals, Inc. Methods and compositions for re-dosing aav using anti-cd40 antagonistic antibody to suppress host anti-aav antibody response
WO2025202473A1 (en) 2024-03-28 2025-10-02 Revvity Discovery Limited A nucleic acid deaminase, a base editor and uses thereof
WO2025235388A1 (en) 2024-05-06 2025-11-13 Regeneron Pharmaceuticals, Inc. Transgene genomic identification by nuclease-mediated long read sequencing
WO2025240946A1 (en) 2024-05-17 2025-11-20 Intellia Therapeutics, Inc. Lipid nanoparticles and lipid nanoparticle compositions
WO2025250454A1 (en) 2024-05-28 2025-12-04 University Of Rochester Adeno-associated viruses evolved to specifically target human glial progenitor cells in vivo
WO2025255308A1 (en) 2024-06-07 2025-12-11 Intellia Therapeutics, Inc. Cd8 co-receptor chimeric polypeptides in tcr cell therapy
WO2025260068A1 (en) 2024-06-14 2025-12-18 Tune Therapeutics, Inc. Lipid nanoparticle formulation for delivery of nucleic acids to cells

Citations (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20070020627A1 (en) 2002-06-11 2007-01-25 The Scripps Research Institute Artificial transcription factors
US20110236894A1 (en) 2008-09-26 2011-09-29 Immune Disease Institute, Inc. Selective oxidation of 5-methylcytosine by tet-family proteins

Family Cites Families (164)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US4603044A (en) 1983-01-06 1986-07-29 Technology Unlimited, Inc. Hepatocyte Directed Vesicle delivery system
US4957773A (en) 1989-02-13 1990-09-18 Syracuse University Deposition of boron-containing films from decaborane
US5436150A (en) * 1992-04-03 1995-07-25 The Johns Hopkins University Functional domains in flavobacterium okeanokoities (foki) restriction endonuclease
JP4118327B2 (ja) 1994-08-20 2008-07-16 ゲンダック・リミテッド Ｄｎａ認識のための結合タンパク質におけるまたはそれに関連する改良
US20030017149A1 (en) 1996-10-10 2003-01-23 Hoeffler James P. Single chain monoclonal antibody fusion reagents that regulate transcription in vivo
US6534261B1 (en) 1999-01-12 2003-03-18 Sangamo Biosciences, Inc. Regulation of endogenous gene expression in cells using zinc finger proteins
US20020164575A1 (en) 1999-09-14 2002-11-07 Sangamo Biosciences, Inc., A Delaware Corporation Gene identification
DE60023936T2 (de) 1999-12-06 2006-05-24 Sangamo Biosciences Inc., Richmond Methoden zur verwendung von randomisierten zinkfingerprotein-bibliotheken zur identifizierung von genfunktionen
ATE353361T1 (de) 2000-04-28 2007-02-15 Sangamo Biosciences Inc Gezielten modifikation der chromatinstruktur
AU2001257331A1 (en) 2000-04-28 2001-11-12 Sangamo Biosciences, Inc. Methods for designing exogenous regulatory molecules
US20030198627A1 (en) 2001-09-01 2003-10-23 Gert-Jan Arts siRNA knockout assay method and constructs
WO2003072788A1 (en) 2002-02-21 2003-09-04 The Wistar Institute Of Anatomy And Biology Methods and compositions for reversibly controlling expression of target genes in cells
WO2004099366A2 (en) 2002-10-23 2004-11-18 The General Hospital Corporation Context sensitive parallel optimization of zinc finger dna binding domains
US7021555B2 (en) 2004-01-06 2006-04-04 Zoo Med Laboratories, Inc. Spraying/misting for plants and animals
US7919277B2 (en) 2004-04-28 2011-04-05 Danisco A/S Detection and typing of bacterial strains
SI2351772T1 (sl) 2005-02-18 2016-11-30 Glaxosmithkline Biologicals Sa Proteini in nukleinske kisline iz Escherichia coli povezane z meningitisom/sepso
US20100055793A1 (en) 2005-07-25 2010-03-04 Johns Hopkins University Site-specific modification of the human genome using custom-designed zinc finger nucleases
WO2007014275A2 (en) 2005-07-26 2007-02-01 Sangamo Biosciences, Inc. Targeted integration and expression of exogenous nucleic acid sequences
DK2341149T3 (en) 2005-08-26 2017-02-27 Dupont Nutrition Biosci Aps Use of CRISPR-associated genes (Cas)
JP2009520463A (ja) 2005-11-28 2009-05-28 ザ スクリプス リサーチ インスティテュート Ｔｎｎのための亜鉛フィンガー結合ドメイン
EP2426220B1 (en) 2006-05-19 2016-06-22 DuPont Nutrition Biosciences ApS Tagged microorganisms and methods of tagging
JP5266210B2 (ja) 2006-05-25 2013-08-21 サンガモ バイオサイエンシズ インコーポレイテッド 改変開裂ハーフドメイン
EP2034848B1 (en) 2006-06-16 2016-10-19 DuPont Nutrition Biosciences ApS Streptococcus thermophilus bacterium
US9201063B2 (en) 2006-11-16 2015-12-01 General Electric Company Sequential analysis of biological samples
WO2008093152A1 (en) * 2007-02-01 2008-08-07 Cellectis Obligate heterodimer meganucleases and uses thereof
RU2531343C2 (ru) 2007-03-02 2014-10-20 ДюПон Ньютришн Байосайенсиз АпС, Способ генерирования заквасочной культуры, заквасочная культура и способ ферментации с ее использованием
WO2008118394A1 (en) 2007-03-23 2008-10-02 New York University Methods of affecting nitrogen assimilation in plants
US8252535B2 (en) 2007-04-10 2012-08-28 Qiagen Gmbh RNA interference tags
WO2008151032A2 (en) 2007-05-31 2008-12-11 Washington University In St. Louis Arrays and methods comprising m. smithii gene products
BRPI0817299A8 (pt) 2007-09-25 2019-01-29 Pastoral Greenhouse Gas Res Limited vacinas e componentes de vacina para inibição de células microbianas
FR2925918A1 (fr) 2007-12-28 2009-07-03 Pasteur Institut Typage et sous-typage moleculaire de salmonella par identification des sequences nucleotidiques variables des loci crispr
FR2930264B1 (fr) 2008-04-18 2013-02-22 Gervais Danone Sa Nouvelle souche de lactobacillus paracasei subsp. paracasei dotee de proprietes antimicrobiennes et immunomodulatrices.
JP2010017179A (ja) 2008-06-11 2010-01-28 Sumitomo Chemical Co Ltd Ｄｎａを定量又は検出する方法
JP2010017178A (ja) 2008-06-11 2010-01-28 Sumitomo Chemical Co Ltd Ｄｎａを定量又は検出する方法
WO2010011961A2 (en) 2008-07-25 2010-01-28 University Of Georgia Research Foundation, Inc. Prokaryotic rnai-like system and methods of use
JP2010048566A (ja) 2008-08-19 2010-03-04 Sumitomo Chemical Co Ltd Ｄｎａを定量又は検出する方法
JP2010068800A (ja) 2008-08-19 2010-04-02 Sumitomo Chemical Co Ltd Ｄｎａを定量又は検出する方法
US20100076057A1 (en) 2008-09-23 2010-03-25 Northwestern University TARGET DNA INTERFERENCE WITH crRNA
NZ592994A (en) 2008-10-21 2012-12-21 Animal Health Trust Diagnostic test for Streptococcus equi comprising assessing the presence or absence of the S. equi eqbE gene
WO2010046493A2 (en) 2008-10-23 2010-04-29 Université de Lausanne Gene transfer vectors comprising at least one isolated dna molecule having insulator and or boundary properties and methods to identify the same
US9404098B2 (en) 2008-11-06 2016-08-02 University Of Georgia Research Foundation, Inc. Method for cleaving a target RNA using a Cas6 polypeptide
MX337838B (es) * 2008-11-07 2016-03-22 Dupont Nutrition Biosci Aps Secuencias de repetidos palindromicos cortos regularmente intercalados agrupados de bifidobacterias.
RU2570557C2 (ru) 2008-11-11 2015-12-10 Алиментари Хелс Лимитед ПРОБИОТИЧЕСКАЯ БИФИДОБАКТЕРИЯ Bifidobacterium Longum
GB2466177A (en) 2008-12-03 2010-06-16 Arab Science & Technology Found Bacteriophage selection and breeding
WO2010066907A1 (en) 2008-12-12 2010-06-17 Danisco A/S Genetic cluster of strains of streptococcus thermophilus having unique rheological properties for dairy fermentation
KR20100093626A (ko) 2009-02-17 2010-08-26 서강대학교산학협력단 슈도모나스 애루지노사에 대한 파아지 치료
WO2010113037A1 (en) 2009-04-03 2010-10-07 Centre National De La Recherche Scientifique Gene transfer vectors comprising genetic insulator elements and methods to identify genetic insulator elements
US8501405B2 (en) 2009-04-27 2013-08-06 Pacific Biosciences Of California, Inc. Real-time sequencing methods and systems
WO2010144151A2 (en) 2009-06-12 2010-12-16 Pacific Biosciences Of California, Inc. Single-molecule real-time analysis of protein synthesis
US20120178647A1 (en) 2009-08-03 2012-07-12 The General Hospital Corporation Engineering of zinc finger arrays by context-dependent assembly
CA2773879A1 (en) 2009-09-25 2011-03-31 Basf Plant Science Company Gmbh Plants having enhanced yield-related traits and a method for making the same
US9677125B2 (en) 2009-10-21 2017-06-13 General Electric Company Detection of plurality of targets in biological samples
US20110269119A1 (en) 2009-10-30 2011-11-03 Synthetic Genomics, Inc. Encoding text into nucleic acid sequences
CA2788560A1 (en) 2010-02-08 2011-08-11 Sangamo Biosciences, Inc. Engineered cleavage half-domains
WO2011101696A1 (en) * 2010-02-18 2011-08-25 Cellectis Improved meganuclease recombination system
US20120027786A1 (en) 2010-02-23 2012-02-02 Massachusetts Institute Of Technology Genetically programmable pathogen sense and destroy
US10087431B2 (en) 2010-03-10 2018-10-02 The Regents Of The University Of California Methods of generating nucleic acid fragments
US10645934B2 (en) 2010-03-12 2020-05-12 Brookhaven Science Associates/Brookhaven National Laboratory Enterobacter sp-638 and methods of use thereof
CN103038338B (zh) 2010-05-10 2017-03-08 加利福尼亚大学董事会 核糖核酸内切酶组合物及其使用方法
EP2580331A4 (en) 2010-06-14 2013-11-27 Univ Iowa State Res Found Inc NUCLEASE ACTIVITY OF THE TAL EFFECTOR AND FUSION PROTEIN FOKI
WO2012047726A1 (en) 2010-09-29 2012-04-12 The Broad Institute, Inc. Methods for chromatin immuno-precipitations
EA201390586A1 (ru) 2010-10-20 2014-11-28 ДюПон НЬЮТРИШН БАЙОСАЙЕНСИЗ АпС Последовательности crispr-cas lactococcus
SG189482A1 (en) 2010-10-27 2013-05-31 Cellectis Method for increasing the efficiency of double-strand break-induced mutagenesis
KR101556359B1 (ko) * 2011-01-03 2015-10-01 주식회사 툴젠 디자인된 tal 이펙터 뉴클레아제를 통한 게놈 엔지니어링
US20120214160A1 (en) 2011-01-14 2012-08-23 Life Technologies Corporation Methods, compositions, and kits for detecting rare cells
WO2012164565A1 (en) 2011-06-01 2012-12-06 Yeda Research And Development Co. Ltd. Compositions and methods for downregulating prokaryotic genes
DK2543255T4 (da) 2011-07-04 2023-03-20 Dsm Ip Assets Bv Antilisteriel blandet kultur og fremgangsmåde til fremstilling af ost
CA2841710C (en) 2011-07-15 2021-03-16 The General Hospital Corporation Methods of transcription activator like effector assembly
GB201122458D0 (en) 2011-12-30 2012-02-08 Univ Wageningen Modified cascade ribonucleoproteins and uses thereof
SG10201606959PA (en) 2012-02-24 2016-09-29 Hutchinson Fred Cancer Res Compositions and methods for the treatment of hemoglobinopathies
MX374399B (es) 2012-02-29 2025-03-06 Sangamo Biosciences Inc Composiciones y sus usos para tratar y prevenir la enfermedad de huntington.
WO2013141680A1 (en) 2012-03-20 2013-09-26 Vilnius University RNA-DIRECTED DNA CLEAVAGE BY THE Cas9-crRNA COMPLEX
US9637739B2 (en) 2012-03-20 2017-05-02 Vilnius University RNA-directed DNA cleavage by the Cas9-crRNA complex
JP6352250B2 (ja) 2012-05-02 2018-07-04 ダウ アグロサイエンシィズ エルエルシー リンゴ酸デヒドロゲナーゼの標的改変
CA2871524C (en) 2012-05-07 2021-07-27 Sangamo Biosciences, Inc. Methods and compositions for nuclease-mediated targeted integration of transgenes
US11120889B2 (en) 2012-05-09 2021-09-14 Georgia Tech Research Corporation Method for synthesizing a nuclease with reduced off-site cleavage
SI3401400T1 (sl) 2012-05-25 2019-10-30 Univ California Postopki in sestavki za RNA usmerjeno modifikacijo tarčne DNA in za RNA usmerjeno modulacijo prepisovanja
US9102936B2 (en) 2012-06-11 2015-08-11 Agilent Technologies, Inc. Method of adaptor-dimer subtraction using a CRISPR CAS6 protein
EP2674501A1 (en) 2012-06-14 2013-12-18 Agence nationale de sécurité sanitaire de l'alimentation,de l'environnement et du travail Method for detecting and identifying enterohemorrhagic Escherichia coli
US10025647B2 (en) * 2012-06-30 2018-07-17 Intel Corporation Memory poisoning with hints
US10883119B2 (en) 2012-07-11 2021-01-05 Sangamo Therapeutics, Inc. Methods and compositions for delivery of biologics
CN105188767A (zh) 2012-07-25 2015-12-23 布罗德研究所有限公司 可诱导的dna结合蛋白和基因组干扰工具及其应用
HK1217732A1 (zh) 2012-09-07 2017-01-20 美国陶氏益农公司 Fad3性能基因座及相應的能夠誘導靶向斷裂的靶位點特異性結合蛋白
AU2013329308B2 (en) 2012-10-09 2018-11-01 Liposcience, Inc. NMR quantification of branched chain amino acids
EP3789405A1 (en) * 2012-10-12 2021-03-10 The General Hospital Corporation Transcription activator-like effector (tale) - lysine-specific demethylase 1 (lsd1) fusion proteins
WO2014071235A1 (en) 2012-11-01 2014-05-08 Massachusetts Institute Of Technology Genetic device for the controlled destruction of dna
EP3138911B1 (en) * 2012-12-06 2018-12-05 Sigma Aldrich Co. LLC Crispr-based genome modification and regulation
US20140310830A1 (en) 2012-12-12 2014-10-16 Feng Zhang CRISPR-Cas Nickase Systems, Methods And Compositions For Sequence Manipulation in Eukaryotes
ES2701749T3 (es) 2012-12-12 2019-02-25 Broad Inst Inc Métodos, modelos, sistemas y aparatos para identificar secuencias diana para enzimas Cas o sistemas CRISPR-Cas para secuencias diana y transmitir resultados de los mismos
US8697359B1 (en) 2012-12-12 2014-04-15 The Broad Institute, Inc. CRISPR-Cas systems and methods for altering expression of gene products
BR112015013784A2 (pt) 2012-12-12 2017-07-11 Massachusetts Inst Technology aplicação, manipulação e otimização de sistemas, métodos e composições para manipulação de sequência e aplicações terapêuticas
EP3064585B1 (en) 2012-12-12 2020-02-05 The Broad Institute, Inc. Engineering and optimization of improved systems, methods and enzyme compositions for sequence manipulation
CN105658796B (zh) 2012-12-12 2021-10-26 布罗德研究所有限公司 用于序列操纵的crispr-cas组分系统、方法以及组合物
CN113355357B (zh) 2012-12-12 2024-12-03 布罗德研究所有限公司 对用于序列操纵的改进的系统、方法和酶组合物进行的工程化和优化
ES2536353T3 (es) 2012-12-12 2015-05-22 The Broad Institute, Inc. Ingeniería de sistemas, métodos y composiciones de guía optimizadas para manipulación de secuencias
WO2014093701A1 (en) 2012-12-12 2014-06-19 The Broad Institute, Inc. Functional genomics using crispr-cas systems, compositions, methods, knock out libraries and applications thereof
EP2931898B1 (en) 2012-12-12 2016-03-09 The Broad Institute, Inc. Engineering and optimization of systems, methods and compositions for sequence manipulation with functional domains
CN105074061B (zh) 2012-12-13 2021-03-09 美国陶氏益农公司 位点特异性核酸酶活性的dna检测方法
DK3553174T3 (da) * 2012-12-17 2025-08-04 Harvard College Rna-guided modificering af humant genom
AR094098A1 (es) * 2012-12-19 2015-07-08 Dow Agrosciences Llc Transformacion de soja mejorada para una produccion eficaz de eventos transgenicos de alto rendimiento
NZ629569A (en) 2013-01-14 2018-07-27 Recombinetics Inc Hornless livestock
US20140212869A1 (en) 2013-01-25 2014-07-31 Agilent Technologies, Inc. Nucleic Acid Proximity Assay Involving the Formation of a Three-way junction
CN103233028B (zh) 2013-01-25 2015-05-13 南京徇齐生物技术有限公司 一种无物种限制无生物安全性问题的真核生物基因打靶方法及螺旋结构dna序列
US10660943B2 (en) 2013-02-07 2020-05-26 The Rockefeller University Sequence specific antimicrobials
EP2954042B1 (en) 2013-02-07 2017-12-06 The General Hospital Corporation Tale transcriptional activators
WO2014127287A1 (en) * 2013-02-14 2014-08-21 Massachusetts Institute Of Technology Method for in vivo tergated mutagenesis
EP2958996B1 (en) 2013-02-25 2019-10-16 Sangamo Therapeutics, Inc. Methods and compositions for enhancing nuclease-mediated gene disruption
EP2922393B2 (en) 2013-02-27 2022-12-28 Helmholtz Zentrum München - Deutsches Forschungszentrum für Gesundheit und Umwelt (GmbH) Gene editing in the oocyte by cas9 nucleases
EP2964779B1 (en) 2013-03-08 2018-08-29 Oxford Nanopore Technologies Limited Use of spacer elements in a nucleic acid to control movement of a helicase
US10612043B2 (en) 2013-03-09 2020-04-07 Agilent Technologies, Inc. Methods of in vivo engineering of large sequences using multiple CRISPR/cas selections of recombineering events
US20140315985A1 (en) 2013-03-14 2014-10-23 Caribou Biosciences, Inc. Compositions and methods of nucleic acid-targeting nucleic acids
US11332719B2 (en) 2013-03-15 2022-05-17 The Broad Institute, Inc. Recombinant virus and preparations thereof
WO2014144155A1 (en) 2013-03-15 2014-09-18 Regents Of The University Of Minnesota Engineering plant genomes using crispr/cas systems
US20140273230A1 (en) 2013-03-15 2014-09-18 Sigma-Aldrich Co., Llc Crispr-based genome modification and regulation
US9234213B2 (en) 2013-03-15 2016-01-12 System Biosciences, Llc Compositions and methods directed to CRISPR/Cas genomic engineering systems
US10760064B2 (en) 2013-03-15 2020-09-01 The General Hospital Corporation RNA-guided targeting of genetic and epigenomic regulatory proteins to specific genomic loci
US9885033B2 (en) 2013-03-15 2018-02-06 The General Hospital Corporation Increasing specificity for RNA-guided genome editing
US20140349400A1 (en) 2013-03-15 2014-11-27 Massachusetts Institute Of Technology Programmable Modification of DNA
UA121197C2 (uk) 2013-04-05 2020-04-27 Доу Агросайенсіс Ллс Нуклеаза "цинкові пальці" для модифікацїї гена ahas та спосіб її використання
US20150056629A1 (en) 2013-04-14 2015-02-26 Katriona Guthrie-Honea Compositions, systems, and methods for detecting a DNA sequence
SG10201808935WA (en) 2013-04-16 2018-11-29 Regeneron Pharma Targeted modification of rat genome
CN103224947B (zh) 2013-04-28 2015-06-10 陕西师范大学 一种基因打靶系统
JP2016518142A (ja) 2013-05-10 2016-06-23 サンガモ バイオサイエンシーズ， インコーポレイテッド ヌクレアーゼ媒介ゲノム遺伝子操作のための送達方法および組成物
US11414695B2 (en) 2013-05-29 2022-08-16 Agilent Technologies, Inc. Nucleic acid enrichment using Cas9
US20150067922A1 (en) 2013-05-30 2015-03-05 The Penn State Research Foundation Gene targeting and genetic modification of plants via rna-guided genome editing
US20150315252A1 (en) 2013-06-11 2015-11-05 Clontech Laboratories, Inc. Protein enriched microvesicles and methods of making and using the same
JP6625971B2 (ja) 2013-06-17 2019-12-25 ザ・ブロード・インスティテュート・インコーポレイテッド 配列操作のためのタンデムガイド系、方法および組成物の送達、エンジニアリングおよび最適化
US10011850B2 (en) 2013-06-21 2018-07-03 The General Hospital Corporation Using RNA-guided FokI Nucleases (RFNs) to increase specificity for RNA-Guided Genome Editing
CN103343120B (zh) 2013-07-04 2015-03-04 中国科学院遗传与发育生物学研究所 一种小麦基因组定点改造方法
JP6482546B2 (ja) 2013-07-19 2019-03-13 ラリクス・バイオサイエンス・リミテッド・ライアビリティ・カンパニーＬａｒｉｘ Ｂｉｏｓｃｉｅｎｃｅ， Ｌｌｃ 二重対立遺伝子ノックアウトを生成するための方法および組成物
WO2015021426A1 (en) 2013-08-09 2015-02-12 Sage Labs, Inc. A crispr/cas system-based novel fusion protein and its application in genome editing
KR102829712B1 (ko) 2013-08-29 2025-07-10 템플 유니버시티-오브 더 커먼웰쓰 시스템 오브 하이어 에듀케이션 Hiv 감염증의 rna-유도 치료를 위한 방법 및 조성물
EP3041931B1 (en) 2013-09-04 2020-06-10 Csir Site-specific nuclease single-cell assay targeting gene regulatory elements to silence gene expression
US9388430B2 (en) 2013-09-06 2016-07-12 President And Fellows Of Harvard College Cas9-recombinase fusion proteins and uses thereof
US9074199B1 (en) 2013-11-19 2015-07-07 President And Fellows Of Harvard College Mutant Cas9 proteins
JP6174811B2 (ja) 2013-12-11 2017-08-02 リジェネロン・ファーマシューティカルズ・インコーポレイテッドＲｅｇｅｎｅｒｏｎ Ｐｈａｒｍａｃｅｕｔｉｃａｌｓ， Ｉｎｃ． ゲノムの標的改変のための方法及び組成物
WO2015089364A1 (en) 2013-12-12 2015-06-18 The Broad Institute Inc. Crystal structure of a crispr-cas system, and uses thereof
JP2017501149A (ja) 2013-12-12 2017-01-12 ザ・ブロード・インスティテュート・インコーポレイテッド 粒子送達構成成分を用いた障害及び疾患の標的化のためのｃｒｉｓｐｒ−ｃａｓ系及び組成物の送達、使用及び治療適用
US20150191744A1 (en) 2013-12-17 2015-07-09 University Of Massachusetts Cas9 effector-mediated regulation of transcription, differentiation and gene editing/labeling
AU2014370416B2 (en) 2013-12-26 2021-03-11 The General Hospital Corporation Multiplex guide RNAs
EP3105327A4 (en) 2014-02-12 2017-10-18 Thomas Jefferson University Compositions and methods of using microrna inhibitors
WO2015138510A1 (en) 2014-03-10 2015-09-17 Editas Medicine., Inc. Crispr/cas-related methods and compositions for treating leber's congenital amaurosis 10 (lca10)
EP3117004A4 (en) 2014-03-14 2017-12-06 University of Washington Genomic insulator elements and uses thereof
US10323073B2 (en) 2014-03-20 2019-06-18 UNIVERSITé LAVAL CRISPR-based methods and products for increasing frataxin levels and uses thereof
CN106170550A (zh) 2014-04-03 2016-11-30 麻省理工学院 用于产生导引rna的方法和组合物
EP4464338A3 (en) 2014-11-07 2025-02-12 Editas Medicine, Inc. Systems for improving crispr/cas-mediated genome-editing
MA41349A (fr) 2015-01-14 2017-11-21 Univ Temple Éradication de l'herpès simplex de type i et d'autres virus de l'herpès associés guidée par arn
SG10201804715WA (en) 2015-01-28 2018-07-30 Pioneer Hi Bred Int Crispr hybrid dna/rna polynucleotides and methods of use
US20190388469A1 (en) 2015-01-30 2019-12-26 The Regents Of The University Of California Protein delivery in primary hematopoietic cells
WO2016130600A2 (en) 2015-02-09 2016-08-18 Duke University Compositions and methods for epigenome editing
WO2016141224A1 (en) 2015-03-03 2016-09-09 The General Hospital Corporation Engineered crispr-cas9 nucleases with altered pam specificity
WO2016191684A1 (en) 2015-05-28 2016-12-01 Finer Mitchell H Genome editing vectors
US9790490B2 (en) 2015-06-18 2017-10-17 The Broad Institute Inc. CRISPR enzymes and systems
WO2016205759A1 (en) 2015-06-18 2016-12-22 The Broad Institute Inc. Engineering and optimization of systems, methods, enzymes and guide scaffolds of cas9 orthologs and variants for sequence manipulation
EP3337908A4 (en) 2015-08-18 2019-01-23 The Broad Institute, Inc. METHOD AND COMPOSITIONS FOR CHANGING THE FUNCTION AND STRUCTURE OF CHROMATIN GRINDING AND / OR DOMAINS
US9926546B2 (en) 2015-08-28 2018-03-27 The General Hospital Corporation Engineered CRISPR-Cas9 nucleases
US9512446B1 (en) 2015-08-28 2016-12-06 The General Hospital Corporation Engineered CRISPR-Cas9 nucleases
KR102668608B1 (ko) 2015-08-28 2024-05-24 더 제너럴 하스피탈 코포레이션 조작된 crispr-cas9 뉴클레아제
CN110290813A (zh) 2016-10-14 2019-09-27 通用医疗公司 表观遗传学调控的位点特异性核酸酶
EP3579858A4 (en) 2017-02-07 2020-12-23 The Regents of The University of California GENE THERAPY AGAINST HAPLOINSUFFICIENCY
JP7379160B2 (ja) 2017-04-21 2023-11-14 ザ ジェネラル ホスピタル コーポレイション ＣＲＩＳＰＲ－Ｃｐｆ１を使用する誘導性で調整可能な多重ヒト遺伝子制御
US11041155B2 (en) 2018-05-17 2021-06-22 The General Hospital Corporation CCCTC-binding factor variants
CA3163087A1 (en) 2019-11-27 2021-06-03 The General Hospital Corporation System and method for activating gene expression
WO2021243289A1 (en) 2020-05-29 2021-12-02 The General Hospital Corporation Systems and methods for stable and heritable alteration by precision editing (shape)

Patent Citations (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20070020627A1 (en) 2002-06-11 2007-01-25 The Scripps Research Institute Artificial transcription factors
US20110236894A1 (en) 2008-09-26 2011-09-29 Immune Disease Institute, Inc. Selective oxidation of 5-methylcytosine by tet-family proteins

Non-Patent Citations (1)

* Cited by examiner, † Cited by third party
Title
Cell, Vol.152, pp.1173-1183 (2013.02.28) 1부.

Also Published As

Publication number Publication date
IL289396A (en) 2022-02-01
ES2713503T3 (es) 2019-05-22
AU2020207840A1 (en) 2020-08-13
EP3467125A1 (en) 2019-04-10
US20200224222A1 (en) 2020-07-16
US10378027B2 (en) 2019-08-13
US11098326B2 (en) 2021-08-24
EP2970986A4 (en) 2016-09-07
IL289396B2 (en) 2023-12-01
AU2020207840B2 (en) 2022-07-21
CN110540991A (zh) 2019-12-06
WO2014144592A3 (en) 2014-12-31
JP6622183B2 (ja) 2019-12-18
EP2971125A4 (en) 2016-08-24
AU2022252788B2 (en) 2025-02-20
AU2020201465B2 (en) 2022-05-12
EP3988667A1 (en) 2022-04-27
JP6657069B2 (ja) 2020-03-04
EP3744842A1 (en) 2020-12-02
US9567603B2 (en) 2017-02-14
JP2020031637A (ja) 2020-03-05
AU2014228981B2 (en) 2019-11-28
US11168338B2 (en) 2021-11-09
KR102874079B1 (ko) 2025-10-22
JP2020039350A (ja) 2020-03-19
EP3741868A1 (en) 2020-11-25
US20180340189A1 (en) 2018-11-29
EP2971125B1 (en) 2020-05-06
KR102210323B1 (ko) 2021-02-01
US11920152B2 (en) 2024-03-05
CN105408497A (zh) 2016-03-16
EP2970208A2 (en) 2016-01-20
AU2014227653A2 (en) 2015-11-12
US20140295557A1 (en) 2014-10-02
US9885033B2 (en) 2018-02-06
AU2014239665A1 (en) 2015-10-01
WO2014144761A3 (en) 2015-10-29
EP4428141A3 (en) 2024-12-18
JP2016517276A (ja) 2016-06-16
US20200071730A1 (en) 2020-03-05
US10138476B2 (en) 2018-11-27
KR20210013303A (ko) 2021-02-03
US11634731B2 (en) 2023-04-25
AU2019204675A1 (en) 2019-07-18
WO2014152432A2 (en) 2014-09-25
EP2970986B1 (en) 2020-05-06
EP2971041A4 (en) 2016-09-07
ZA201506814B (en) 2018-11-28
CN113563476A (zh) 2021-10-29
JP2023061983A (ja) 2023-05-02
BR112015023489B1 (pt) 2022-06-07
US10844403B2 (en) 2020-11-24
EP4428141A2 (en) 2024-09-11
AU2017204909B2 (en) 2019-04-04
KR102405549B1 (ko) 2022-06-08
KR20150132395A (ko) 2015-11-25
JP7126588B2 (ja) 2022-08-26
AU2022209254B2 (en) 2025-01-23
US20210130850A1 (en) 2021-05-06
US20170152508A1 (en) 2017-06-01
AU2022209254A1 (en) 2022-08-18
US20190376090A1 (en) 2019-12-12
CA2906724A1 (en) 2014-09-18
CA2906553C (en) 2022-08-02
KR20150131250A (ko) 2015-11-24
JP6980380B2 (ja) 2021-12-15
JP6878554B2 (ja) 2021-05-26
EP3741868B1 (en) 2024-05-22
US10415059B2 (en) 2019-09-17
CN105247066B (zh) 2020-10-20
KR20210013304A (ko) 2021-02-03
CN110540991B (zh) 2023-10-24
US20230407341A1 (en) 2023-12-21
JP6960438B2 (ja) 2021-11-05
JP2020120661A (ja) 2020-08-13
CN112301024A (zh) 2021-02-02
WO2014144761A2 (en) 2014-09-18
CN105408497B (zh) 2019-09-13
US9567604B2 (en) 2017-02-14
BR112015023489A2 (pt) 2017-10-10
AU2019204675B2 (en) 2021-03-11
US20220090145A1 (en) 2022-03-24
AU2014239665B2 (en) 2020-04-30
AU2014227653B2 (en) 2017-04-20
EP2970208A4 (en) 2016-11-30
US20160010076A1 (en) 2016-01-14
JP2016512264A (ja) 2016-04-25
WO2014152432A3 (en) 2015-10-29
CA2906553A1 (en) 2014-09-25
EP2971041A1 (en) 2016-01-20
US10119133B2 (en) 2018-11-06
KR20210013302A (ko) 2021-02-03
CN105247066A (zh) 2016-01-13
IL289396B1 (en) 2023-08-01
AU2017204909A1 (en) 2017-08-31
CA2907198C (en) 2019-12-10
KR20220080012A (ko) 2022-06-14
KR102271291B1 (ko) 2021-07-02
AU2021203370A1 (en) 2021-06-24
EP3467125B1 (en) 2023-08-30
US20160024523A1 (en) 2016-01-28
EP2971125A2 (en) 2016-01-20
EP2971125B2 (en) 2023-11-22
KR20150131251A (ko) 2015-11-24
CA2907198A1 (en) 2014-09-18
US10544433B2 (en) 2020-01-28
WO2014144288A1 (en) 2014-09-18
KR102210322B1 (ko) 2021-02-01
EP2971041B1 (en) 2018-11-28
AU2021203370B2 (en) 2023-07-27
CA3161835A1 (en) 2014-09-25
US20180208921A1 (en) 2018-07-26
US20140295556A1 (en) 2014-10-02
KR102210319B1 (ko) 2021-02-01
KR20230157540A (ko) 2023-11-16
JP2024012446A (ja) 2024-01-30
JP2021118726A (ja) 2021-08-12
IL241671B (en) 2022-03-01
US20240240207A1 (en) 2024-07-18
US20250034597A1 (en) 2025-01-30
JP7379572B2 (ja) 2023-11-14
KR102602047B1 (ko) 2023-11-15
US20160024524A1 (en) 2016-01-28
JP2022106710A (ja) 2022-07-20
AU2014227653A1 (en) 2015-10-01
AU2014228981A1 (en) 2015-10-01
AU2022252788A1 (en) 2022-11-03
EP2970986A2 (en) 2016-01-20
JP7053706B2 (ja) 2022-04-12
JP2016512691A (ja) 2016-05-09
AU2020201465A1 (en) 2020-03-19
US12065668B2 (en) 2024-08-20
JP2021192624A (ja) 2021-12-23
CN105408483A (zh) 2016-03-16
US20170327805A1 (en) 2017-11-16
EP3865586A1 (en) 2021-08-18
WO2014144592A2 (en) 2014-09-18

Similar Documents

Publication Publication Date Title
KR102271292B1 (ko) Rna-안내 게놈 편집의 특이성을 증가시키기 위한 rna-안내 foki 뉴클레아제(rfn)의 용도
AU2021231074C1 (en) Class II, type V CRISPR systems
KR102598856B1 (ko) 변경된 PAM 특이성을 갖는 조작된 CRISPR-Cas9 뉴클레아제
KR102773642B1 (ko) 열 안정성 cas9 뉴클레아제
US10011850B2 (en) Using RNA-guided FokI Nucleases (RFNs) to increase specificity for RNA-Guided Genome Editing
AU2017339542A1 (en) S. pyogenes Cas9 mutant genes and polypeptides encoded by same
KR20190104344A (ko) 열안정성 cas9 뉴클레아제
AU2014273085A1 (en) New compact scaffold of Cas9 in the type II CRISPR system
JP2023503618A (ja) 遺伝子発現を活性化するためのシステムおよび方法
US20250059568A1 (en) Class ii, type v crispr systems

Legal Events

Date Code Title Description
A107 Divisional application of patent
PA0104 Divisional application for international application

Comment text: Divisional Application for International Patent

Patent event code: PA01041R01D

Patent event date: 20210125

Application number text: 1020157029177

Filing date: 20151014

PA0201 Request for examination
PG1501 Laying open of application
E701 Decision to grant or registration of patent right
PE0701 Decision of registration

Patent event code: PE07011S01D

Comment text: Decision to Grant Registration

Patent event date: 20210330

PR0701 Registration of establishment

Comment text: Registration of Establishment

Patent event date: 20210624

Patent event code: PR07011E01D

PR1002 Payment of registration fee

Payment date: 20210625

End annual number: 3

Start annual number: 1

PG1601 Publication of registration